TERMS OF USE

Effective Date: October 1, 2018

These Terms of Use (“Terms”) govern your access to and use of the websites and online services that we operate and that link to these Terms, as well as our offline services that reference these Terms (collectively, the “Services”).

Please review these Terms carefully before using the Services because they are a legal contract between you and BioMarin Pharmaceutical Inc. (“BioMarin,” “we,” “us,” or “our”). By using the Services, you agree to be bound by and comply with these Terms. We may change these Terms or modify any features of the Services at any time. The most current version of the Terms can be viewed by clicking on the “Terms of Use” link posted through the Services. You accept the Terms by using the Services, and you accept any changes to the Terms by continuing to use the Services after we post the changes.

Privacy Notice

For more information on how BioMarin processes your personal information, and your rights regarding your personal information, please see our Privacy Notice .

Prohibited Conduct

You may not access or use, or attempt to access or use, the Services to take any action that could harm us or any third party, interfere with the operation of the Services, or in a manner that violates any law(s). For example, and without limitation, you may not:

Impersonate any person or entity or falsely state or otherwise misrepresent your affiliation with any person or entity or the origin of any information you provide;
Engage in unauthorized spidering, scraping, or harvesting of content or personal information, or use any other unauthorized automated means to compile information;
Obtain or attempt to gain unauthorized access to other computer systems, materials, information, or any services available on or through the Services;
Use any device, software, or routine to interfere or attempt to interfere with the proper working of the Services or any activity conducted on the Services or attempt to probe, scan, test the vulnerability of, or breach the security of any system or network;
Circumvent, reverse engineer, decipher, decompile, disassemble, decrypt, or otherwise alter or interfere with (or attempt, encourage, or support anyone else’s attempt to engage in such activities) any of the software comprising or in any way making up a part of the Services. The use or distribution of tools designed for compromising security (e.g., password guessing programs, cracking tools or network probing tools) is strictly prohibited;
Take any action that imposes an unreasonable or disproportionately large load on our network or infrastructure;
Upload or otherwise transmit any communication, software, or material that contains a virus or is otherwise harmful to BioMarin’s or its users’ computers or systems;
Send or cause to be sent any communication (including e-mail) to other users without their consent (e.g., “mailbombs” or “spamming”);
Cause damage, embarrassment, or adverse publicity to BioMarin; or
Engage in any other conduct that restricts or inhibits any person from using or enjoying the Services, or that, in our sole judgment, exposes us or any of our users, affiliates, or any other third party to any liability, damages, or detriment of any type.

Violations of system or network security may result in civil or criminal liability. We may investigate and work with law enforcement authorities to prosecute users who violate the Terms. We may suspend or terminate your access to the Services for any or no reason at any time without notice.

Medical Disclaimers

The Services and any information or content provided therein are for informational and educational purposes only and are not a substitute for the professional judgment of a health care professional in diagnosing and treating patients. BioMarin does not give medical advice, nor does it provide medical or diagnostic services. Your reliance upon any content or information provided through the Services is solely at your own risk.

User Content and Communications

You remain fully responsible for the materials that you provide to us, including, without limitation, information, stories, requests, creative works, pictures, photographs, letters, documents, demos, ideas, suggestions, reviews, concepts, methods, systems, designs, plans, techniques, or other materials submitted, posted, uploaded, sent, or otherwise transmitted to us (“User Content”). You agree not to provide User Content that:

Infringes on the copyright, trademark, patent, or other intellectual property right of any third party;
Is false, misleading, libelous, defamatory, obscene, abusive, hateful, or sexually-explicit;
Violates a third party’s right to privacy or publicity;
Degrades others on the basis of gender, race, class, ethnicity, national origin, religion, sexual preference, disability, or other classification;
Contains epithets or other language or material intended to intimidate or to incite violence;
Contains a virus, worm, Trojan Horse, time bomb, or any other harmful program or component;
Contains any commercial material or solicits any funds (charitable or commercial), perpetuates chain letters or pyramid schemes, promotes commercial entities, or otherwise engages in commercial activity; or
Violates any applicable local, state, national, or international law, or advocates illegal activity.

You may only post User Content that is original and that you have the right to post. By submitting User Content, you grant us a royalty-free, irrevocable, perpetual, non-exclusive, worldwide, fully sublicensable license to publish, reproduce, distribute, display, perform, edit, adapt, modify, translate, create derivative works from, make, sell, export, and otherwise use your User Content (or any portion thereof) in any way that we want and in any form, media, or technology now known or later developed. You hereby waive any moral rights you may have in your User Content. You represent that you have obtained all necessary permissions, including the right to use an individual’s likeness in our advertising and marketing activities, from any individuals identified in or implicated by your submission (including those shown in photographic content), and, in the case of minors, also from their parents or legal guardians, as appropriate.

We are not obligated to publish or use your User Content. BioMarin is not in any manner endorsing the User Content and cannot, and will not, vouch for its reliability. We do not guarantee any confidentiality with respect to any User Content. Please do not send us any confidential or proprietary information or material.

BioMarin is not responsible for any User Content and has no duty to monitor the User Content posted on the Services. You use any information contained in User Content at your own risk.

BioMarin and its designees have the right, in their sole discretion, to monitor, review, edit, remove, delete, disable, refuse, restrict, or terminate access to your User Content or the Services (in whole or in part) at any time, without prior notice and in our sole discretion, for any or no reason. The obligations that you have to us under these Terms shall survive termination of the Services, any use by you of the Services, any User Content on the Services, or these Terms. You will not continue to post any User Content that BioMarin has previously advised you not to post.

Eligibility and Registration

You may be asked to register for certain activities in connection with the Services by creating a user profile. This registration may allow you to participate in interactive features of the Services, such as forums or discussion boards or to enter sweepstakes and contests. When you register, you agree to provide accurate, current, and complete information about yourself as requested or directed on the Services and to promptly update this information to maintain its accuracy. BioMarin has the right to suspend or terminate any account or other registration and to refuse any and all current or future use of the Services if it suspects that such information is inaccurate or incomplete. You are responsible for maintaining the confidentiality of any password and username that you are given or select in connection with the Services, and you are responsible for all activities that occur under your password or account.

Intellectual Property

The Services are protected under the copyright, trademark, and other intellectual property laws of the United States and other countries. All intellectual property rights in the Services are owned by us or our third-party licensors to the full extent permitted under U.S. and international intellectual property laws. Except for content that you have posted on the Services, you may not publish, reproduce, distribute, display, perform, edit, adapt, modify, or otherwise exploit any part of the Services, including the BioMarin name and logo, without our written consent. You may provide a link to the top page of the Services unless and until BioMarin gives notice that you must discontinue linking to the Services.

We respect intellectual property rights. If you believe in good faith that your work has been reproduced or is accessible on the Services in a way that constitutes copyright infringement, please provide our designated agent with the following information in writing:

An electronic or physical signature of the person authorized to act on behalf of the owner of the exclusive right that is allegedly infringed;
Identification of the copyrighted work or a representative list of the works claimed to have been infringed;
Identification of the allegedly infringing material and information reasonably sufficient to permit us to locate the material;
Your name, address, telephone number, and email address, so that BioMarin may contact you if necessary;
A statement that you have a good faith belief that the disputed use is not authorized by the copyright owner, its agent, or the law; and
A statement by you, made under penalty of perjury, that the above information in your notice is accurate and that you are the copyright owner or authorized to act on the copyright owner's behalf.

BioMarin’s designated agent for notice of claims of alleged copyright infringement is:

Eric Davis
Copyright Agent
105 Digital Drive
Novato, CA 94949
Phone: (415) 506-6700
edavis@bmrn.com

If you believe that a user of the Services is a repeat infringer, please contact BioMarin’s designated agent, as described above, and provide enough information for us to verify that the individual is a repeat infringer. Please note that United States law provides significant penalties for falsely submitting a notice of copyright infringement.

Third-Party Content and Links to Third-Party Websites

The Services may contain links to third-party content. We do not control, endorse, sponsor, recommend, or otherwise accept responsibility for such content. Use of any linked third-party content is at the user's own risk.

Disclaimer of Warranties

YOUR USE OF THE SERVICES IS AT YOUR OWN RISK. WE MAKE NO REPRESENTATIONS OR WARRANTIES ABOUT THE OPERATION OF THE SERVCES OR THE INFORMATION, MATERIALS, GOODS, OR SERVICES APPEARING OR OFFERED ON THE SERVICES, ALL OF WHICH ARE PROVIDED "AS IS."

WITHOUT LIMITING THE GENERALITY OF THE FOREGOING, WE DISCLAIM ALL WARRANTIES, EXPRESS OR IMPLIED, INCLUDING, BUT NOT LIMITED TO, ANY (1) WARRANTIES OF MERCHANTABILITY AND FITNESS FOR A PARTICULAR PURPOSE; (2) WARRANTIES AGAINST INFRINGEMENT OF ANY THIRD-PARTY INTELLECTUAL PROPERTY OR PROPRIETARY RIGHTS; (3) WARRANTIES RELATING TO THE TRANSMISSION OR DELIVERY OF THE SERVICES; (4) WARRANTIES RELATING TO THE ACCURACY, RELIABILITY, CORRECTNESS, OR COMPLETENESS OF DATA MADE AVAILABLE ON THE SERVICES OR OTHERWISE BY BIOMARIN; (5) WARRANTIES OTHERWISE RELATING TO PERFORMANCE, NONPERFORMANCE, OR OTHER ACTS OR OMISSIONS BY BIOMARIN OR ANY THIRD PARTY; AND (6) WARRANTY OF TITLE. FURTHER, THERE IS NO WARRANTY THAT THE SERVICES WILL MEET YOUR NEEDS OR REQUIREMENTS OR THE NEEDS OR REQUIREMENTS OF ANY OTHER PERSON.

WE MAKE NO WARRANTIES, EXPRESS OR IMPLIED, (1) THAT THE SERVICES OR ANY EMAIL WE SEND YOU IS FREE OF VIRUSES OR OTHER HARMFUL COMPONENTS THAT MAY INFECT YOUR COMPUTER EQUIPMENT OR OTHER PROPERTY BECAUSE OF YOUR ACCESS TO, USE OF, OR BROWSING ON THE SERVICES OR YOUR DOWNLOADING OF ANY MATERIALS, DATA, TEXT, IMAGES, VIDEO, OR AUDIO FROM THE SERVICES; OR (2) THAT THE SERVICE, WEBSITE CONTENT, FUNCTIONS, OR MATERIALS CONTAINED THEREIN WILL BE TIMELY, SECURE, ACCURATE, COMPLETE, UP-TO-DATE, OR UNINTERRUPTED. BIOMARIN DOES NOT NECESSARILY ENDORSE, SUPPORT, SANCTION, ENCOURAGE, OR AGREE WITH ANY WEBSITE CONTENT OR ANY SUBMITTED MATERIALS, AND WE EXPRESSLY DISCLAIM ANY AND ALL LIABILITY IN CONNECTION WITH USER CONTENT, INCLUDING LINKS TO OR CONTENT CONTAINED ON THIRD-PARTY WEB SITES.

IF APPLICABLE LAW DOES NOT ALLOW THE EXCLUSION OF SOME OR ALL OF THE ABOVE WARRANTIES TO APPLY TO YOU, THE ABOVE EXCLUSIONS WILL APPLY TO YOU TO THE FULLEST EXTENT PERMITTED BY APPLICABLE LAW.

Limitation of Liability

UNDER NO CIRCUMSTANCES, INCLUDING NEGLIGENCE, WILL BIOMARIN, ITS AFFILIATES, OR ANY PARTY INVOLVED IN CREATING, PRODUCING, OR DELIVERING THE SERVICES BE LIABLE FOR DAMAGES OR LOSSES, INCLUDING, BUT NOT LIMITED TO, DIRECT, INCIDENTAL, CONSEQUENTIAL, INDIRECT, SPECIAL, OR PUNITIVE DAMAGES AND LOST PROFITS, ARISING OUT OF YOUR ACCESS, USE, MISUSE, OR INABILITY TO USE THE SERVICES, WEBSITE CONTENT, USER CONTENT, OR ANY LINKED SITES, HOWEVER CAUSED, WHETHER IN CONTRACT, TORT, NEGLIGENCE, STRICT LIABILITY, OR OTHERWISE, EVEN IF BIOMARIN HAS BEEN ADVISED OF THE POSSIBILITY OF SUCH DAMAGES, OR IN CONNECTION WITH ANY FAILURE OF PERFORMANCE, ERROR, OMISSION, INTERRUPTION, DEFECT, DELAY IN OPERATION OR TRANSMISSION, COMPUTER VIRUS, OR LINE OR SYSTEM FAILURE. BIOMARIN WILL NOT BE A PARTY TO, AND WILL HAVE NO RESPONSIBILITY OR LIABILITY FOR, ANY TRANSACTION NEGOTIATED OR ARRANGED BY A USER ARISING IN CONNECTION WITH THE SERVICES. IN THE EVENT THAT YOU HAVE A DISPUTE WITH ANOTHER USER RELATED TO, ARISING FROM, OR IN ANY WAY CONNECTED WITH USE OF THE SERVICES, YOU RELEASE BIOMARIN FROM ANY CLAIMS, DEMANDS, AND DAMAGES OF EVERY KIND AND NATURE ARISING OUT OF OR IN ANY WAY CONNECTED WITH SUCH A DISPUTE. BIOMARIN HAS NO OBLIGATION TO BECOME INVOLVED IN ANY DISPUTE BETWEEN A USER AND ANY OTHER PERSON.

THESE LIMITATIONS APPLY WHETHER THE ALLEGED LIABILITY IS BASED ON CONTRACT, TORT, NEGLIGENCE, STRICT LIABILITY, OR ANY OTHER BASIS, EVEN IF BIOMARIN HAS BEEN ADVISED OF THE POSSIBILITY OF SUCH DAMAGE. BECAUSE SOME JURISDICTIONS DO NOT ALLOW THE EXCLUSION OR LIMITATION OF INCIDENTAL OR CONSEQUENTIAL DAMAGES, BIOMARIN’S LIABILITY IN SUCH JURISDICTIONS SHALL BE LIMITED TO THE EXTENT PERMITTED BY LAW. WITHOUT LIMITING THE GENERALITY OF THE FOREGOING, BIOMARIN DISCLAIMS ALL LIABILITY OF ANY KIND ARISING FROM THE UNAUTHORIZED ACCESS TO OR USE OF YOUR INFORMATION.

IF YOU ARE DISSATISFIED WITH THE SERVICES, YOUR SOLE REMEDY IS TO STOP USING THE SERVICES.

Indemnification

You agree to defend, indemnify, and hold harmless BioMarin and its affiliates, subsidiaries, parent companies, successors, officers, directors, employees, contractors, services providers, and agents from and against any and all demands, claims, damages, liabilities, judgments, losses, costs, expenses, and harms, including, but not limited to, reasonable attorneys’ fees, arising in connection with your use of the Services (including, without limitation, your User Content), online conduct, any violation of these Terms, or dealings or transactions with other persons resulting from use of the Services.

Applicable Law; Jurisdiction

These Terms are governed by, and must be construed in accordance with, the laws of the United States and the State of California, as applicable, without giving effect to their principles of conflicts of law. By using the Services, you waive any claims that may arise under the laws of other countries or territories.

With respect to any and all disputes arising out of or in connection with the Services or these Terms (including, without limitation, the Privacy Notice), BioMarin and you agree to negotiate in good faith and undertake reasonable efforts to cooperate with one another in order to achieve a mutually satisfactory resolution. If you and BioMarin do not resolve any dispute by informal negotiation, any other effort to resolve the dispute will be conducted exclusively by binding arbitration as described in this section. You are giving up the right to litigate (or participate in as a party or class member) all disputes in court before a judge or jury. Instead, all disputes will be resolved before a neutral arbitrator, whose decision will be final except for a limited right of appeal under the Federal Arbitration Act. Any court with jurisdiction over the parties may enforce the arbitrator’s award.

Any proceedings to resolve or litigate any dispute in any forum will be conducted solely on an individual basis. Neither you nor BioMarin will seek to have any dispute heard as a class action or in any other proceeding in which either party acts or proposes to act in a representative capacity. No arbitration or proceeding will be combined with another without the prior written consent of all parties to all affected arbitrations or proceedings.

BioMarin and you agree that all disputes arising under these Terms that cannot be settled through informal negotiation will be settled exclusively through confidential binding arbitration in San Francisco, California, or another forum mutually agreed upon by the parties, pursuant to the Rules of Arbitration (“Rules”) of the International Chamber of Commerce (“ICC”) by a sole arbitrator nominated by agreement of the parties and confirmed in accordance with said Rules. The arbitrator's award shall be binding and may be entered as a judgment in a court of competent jurisdiction. You agree that BioMarin is entitled to obtain preliminary injunctive relief to the extent allowed by law to enforce any of the terms of these Terms pending a final arbitral decision.

To the extent permitted by law, any claim or dispute under this agreement must be filed within one year in an arbitration proceeding. The one-year period begins when the claim or notice of dispute first could be filed. If a claim or dispute isn't filed within one year, it is permanently barred.

If any other provision of this section is found to be illegal or unenforceable, that provision will be severed, with the remainder of this section remaining in full force and effect.

Separate Terms and Conditions

In connection with your use of the Services, you may be asked to consent to policies or terms and conditions in addition to these Terms. Please read these supplemental policies and terms carefully before making any use of such portions of the Services. Any supplemental terms will not vary or replace these Terms regarding any use of the Services, unless otherwise expressly stated.

Access Outside the United States

BioMarin makes no claim that the Services are appropriate for access or use outside the U.S. Your access and use of the Services outside the U.S. are at your own risk, and you are responsible for compliance with the laws of your jurisdiction.

Miscellaneous

These Terms constitute the entire agreement between BioMarin and you, superseding any prior or contemporaneous communications and proposals (whether oral, written or electronic) between you and us. In the event any provision of these Terms is held unenforceable, it will not affect the validity or enforceability of the remaining provisions and will be replaced by an enforceable provision that comes closest to the intention underlying the unenforceable provision. You agree that no joint venture, partnership, employment, or agency relationship exists between you and BioMarin as a result of these Terms or your access to and use of the Services.

Our failure to enforce any provisions of these Terms or respond to a violation by any party does not waive our right to subsequently enforce any terms or conditions of the Terms or respond to any violations. Nothing contained in these Terms is in derogation of our right to comply with governmental, court, and law enforcement requests or requirements relating to your use of the Services or information provided to or gathered by us with respect to such use.

Contact Information

If you believe that someone has violated these Terms, if you have questions regarding these terms, or if you have questions regarding the Services, please notify us by completing the form found here or by telephone at (415) 506-6700.

Privacy Policy and Notice of Information Practices

Effective Date: October 1, 2018

BioMarin Pharmaceutical Inc. ("BioMarin," “we,” “us,” or “our”) respects the privacy of visitors to our websites and online services and values the confidence of our customers, partners, patients, and employees.

This Privacy Policy and Notice of Information Practices (“Privacy Notice”) sets forth BioMarin’s practices regarding the collection, use, and disclosure of information that you may provide through the websites and online services that we operate and that link to this Privacy Notice, as well as through any of our offline services that reference this Privacy Notice (collectively, the “Services”). Please read the entire Privacy Notice before using our Services.

Collection of Information

We may ask you for some or all of the following types of information when you register with our Services, access various content or features, submit photos and other content, or directly contact us with questions or feedback:

Contact information, such as name, email address, postal address, and telephone number;
User name and password;
Demographic information, such as age information and gender;
Communications preferences;
Search queries;
Stories, comments, photos, and other information posted in our interactive online features; and
Correspondence and other information that you send to us.

We also may collect certain information automatically when you visit the Services, including:

Your browser type and operating system;
Your Internet Protocol (IP) address, which is the number automatically assigned to your computer whenever you access the Internet and that can sometimes be used to derive your general geographic area;
Other unique identifiers, including mobile device identification numbers;
Sites you visited before and after visiting the Services;
Pages you view and links you click on within the Services;
Information collected through cookies, web beacons, and other technologies;
Information about your interactions with email messages, such as the links clicked on and whether the messages were opened or forwarded; and
Standard server log information.

We may use cookies, pixel tags, and similar technologies to automatically collect this information. Cookies are small bits of information that are stored by your computer’s web browser. Pixel tags are very small images or small pieces of data embedded in images, also known as “web beacons” or “clear GIFs,” that can recognize cookies, the time and date a page is viewed, a description of the page where the pixel tag is placed, and similar information from your computer or device. You can decide if and how your computer will accept a cookie by configuring your preferences or options in your browser. However, if you choose to reject cookies, you may not be able to use certain online products, services or features on the Services.

Response to “Do Not Track” Signals

Some Internet browsers include the ability to transmit “Do Not Track” signals. Since uniform standards for “Do Not Track” signals have not yet been adopted, BioMarin does not process or respond to “Do Not Track” signals.

Use of Information

Our legal basis to process your information includes processing that is necessary for legitimate business interests, including but not limited to the following examples:

Providing you with the products, promotions, services, and information you request;
Contacting you via email and otherwise about products, services, and events that we think might be of interest to you;
Maintaining or administering the Services, performing business analyses, or for other internal purposes to improve the quality of our business, the Services, and other products and services we offer;
Publishing stories, comments, photos, and other information posted in our interactive online features;
Processing employment applications and inquiries; and
Customizing and personalizing your use of the Services.

We may also process your information in order to comply with our legal obligations, perform a contract between us and you, establish and defend any legal claims, and in some cases our basis for processing will be because you have consented to our use of your information.

Sharing of Information

We are committed to maintaining your trust, and we want you to understand when and with whom we may share the information we collect.

Corporate Parents and Affiliates. We may share your information with our corporate parents and other affiliated entities for a variety of purposes, including business, operational, and marketing purposes. For more information on those entities, see www.biomarin.com/contact.

Service Providers. We may share your information with service providers that perform certain functions or services on our behalf pursuant to the purposes set out in this Privacy Notice (such as to host the Services, manage databases, perform analyses, or send communications for us).

Other Parties When Required By Law or as Necessary to Protect the Services. We may disclose your information to third parties in order to: protect the legal rights, safety, and security of BioMarin, our corporate parents and affiliates, and the users of our Services; enforce our Terms of Use; prevent fraud (or for risk management purposes); and comply with or respond to law enforcement, or legal process, or a request for cooperation by a government entity, whether or not legally required.

In Connection With a Transfer of Assets. If we sell all or part of our business, or make a sale or transfer of assets, or are otherwise involved in a merger or business transfer, or in the event of bankruptcy, we may transfer your information to one or more third parties as part of that transaction.

Other Parties With Your Consent. We may disclose to third parties information that does not describe or identify individual users, such as aggregate website usage data or demographic reports.

Aggregate Information. We may disclose to third parties information that does not describe or identify individual users, such as aggregate website usage data or demographic reports.

We may allow third parties to place and read their own cookies, web beacons, Local Shared Objects, and similar technologies to collect information through the Services. For example, our third-party service providers may use these technologies to collect information that helps us with traffic measurement, research, and analytics. Local Shared Objects (sometimes referred to as “Flash Cookies”) are similar to standard cookies except that they can be larger and are downloaded to a computer or mobile device by the Adobe Flash media player. Please note that you may need to take additional steps beyond changing your browser settings to refuse or disable Local Shared Objects and similar technologies. For example, Local Shared Objects can be controlled through the instructions on Adobe’s Setting Manager page. If you choose to refuse, disable, or delete these technologies, some of the functionality of the Services may no longer be available to you.

Social Networking Services

BioMarin has worked with certain third-party social media providers to offer you their social networking services through our Services. For example, you can use third-party social networking services, including but not limited to Facebook, Twitter, and others, to share information about your experience on our Services with your friends and followers on those social networking services. These social networking services may be able to collect information about you, including your activity on our Services. These third-party social networking services also may notify your friends, both on our Services and on the social networking services themselves, that you are a user of our Services or about your use of our Services, in accordance with applicable law and their own privacy policies. If you choose to access or make use of third-party social networking services, we may receive information about you that you have made available to those social networking services, including information about your contacts on those social networking services.

You also may be able to link an account from a social networking service (e.g., Facebook, Twitter) to an account through our Services. This may allow you to use your credentials from the other site or service to sign in to certain features on our Services. If you link your account from a third-party site or service, we may collect information from those third-party accounts, and any information that we collect will be governed by this Privacy Policy.

Security and Retention

We maintain reasonable security procedures to help protect against loss, misuse or unauthorized access, disclosure, alteration or destruction of the information you provide through the Services. However, no data transmission over the Internet or stored on a server can be guaranteed to be 100% secure. As a result, while we strive to protect your information and privacy, we cannot guarantee or warrant the security of any information you disclose or transmit to us online and cannot be responsible for the theft, destruction, or inadvertent disclosure of your information. In the event that we believe that the security of your information may have been compromised, we will endeavor to give you appropriate notice as quickly as possible, including by email, and in accordance with applicable laws.

You are responsible for maintaining the confidentiality of your account password and for any access to or use of the Services using your password, whether or not authorized by you. Please notify us immediately of any unauthorized use of your password or account or any other breach of security.

Your information will be retained only for so long as reasonably necessary for the purposes set forth in this Privacy Notice, in accordance with applicable laws.

Children’s Privacy

BioMarin respects the privacy of children, and we are committed to complying with applicable legislation, including the US Children’s Online Privacy Protection Act (COPPA) and the EU General Data Protection Act (GDPR). The Services do not knowingly collect, use, or disclose personal information from children without prior parental consent, except as permitted by applicable legislation.

If you have questions concerning our information practices with respect to children, please contact us using the form located here.

Your rights, depending on where you live, may include the right to review, have deleted, or stop the further collection of your child’s personal information. If you would like to make such a request, please contact us using the form located here.

Your Choices and Your Information

If at any time you wish to stop receiving emails or other communications from us, or if you have submitted information through the Services and would like to have that information deleted from our records, please use the form found here to notify us.

Your rights, depending on where you live, may include the right to object to or request restriction of processing of your personal information, and request access to, erasure and/or portability of your information held by BioMarin, and you may make such a request by contacting us by completing the form found here or by telephone at (415) 506-6700.

You may also have the right to lodge a complaint with the relevant data protection supervisory authority if you believe that our use of your information violates applicable law.

International Transfers

Some of the parties to whom we disclose your information are located in countries that may not provide the same level of data protection as your home country. We take appropriate steps to ensure that such recipients of your information are bound to duties of confidentiality, and BioMarin implements measures such as the EU standard data protection contractual clauses to ensure that any transferred personal information remains protected and secure.

Links to Third-Party Content

As a convenience to our visitors, the Services may link to a number of sites, services, and other content that are operated and maintained by third parties. These third parties operate independently from us, and we do not control their privacy practices. Such links do not constitute an endorsement by BioMarin of the content or the persons or entities associated therewith. This Privacy Notice does not apply to third-party content. We encourage you to review the privacy policies of any third party to whom you provide information.

Policy Updates

This Privacy Notice may be revised from time to time as we add new features and services, as laws change, and as industry privacy and security best practices evolve. We display an effective date on the policy in the upper left corner of this Privacy Policy so that it will be easier for you to know when there has been a change. If we make any material change to this Privacy Notice regarding use or disclosure of personal information, we will provide advance notice through the Services. Small changes or changes that do not significantly affect individual privacy interests may be made at any time and without prior notice.

Contact Us

If you have any questions about this Privacy Notice, you may contact us using the form found here or at:

BioMarin Pharmaceutical Inc.
105 Digital Drive
Novato, CA 94949
Phone: (415) 506-6700

For North American Healthcare Professionals

8 years to diagnosis
Alden, 11

Height: 5 ft, 3 in (160 cm)

Undiagnosed skeletal dysplasia, inconsistent with above-average height
Diagnosed with Maroteaux-Lamy syndrome (MPS VI)

Learn about a no-charge test ›
For North American Healthcare Professionals

18 years to diagnosis
Sara, 27

Height: 4 ft, 7.5 in (141 cm)

Presented with abnormal gait at age 8 and diagnosed with SED; multiple surgeries and health challenges throughout her life

Met with a genetic counselor during pregnancy
Diagnosed with Morquio A (MPS IVA)

Learn about a no-charge test ›
For North American Healthcare Professionals

~ 2 years to diagnosis
Mariah, 8

Height: 4 ft (122 cm)

Presented with hip pain and frequent falls at age 4; diagnosed with bilateral Legg-Calvé-Perthes at age 5 based on X-rays and an MRI
Diagnosed with Morquio A (MPS IVA)

Learn about a no-charge test ›

COULD MPS BE HIDING IN YOUR PRACTICE?

Diverse presentations of MPS can complicate patient identification^1-4

MUCOPOLYSACCHARIDOSIS (MPS) OVERVIEW

A heterogeneous group of rare inherited disorders with several subtypes: MPS I, II, III A-D, IV A/B, VI, VII, or IX.⁵

MPS is caused by enzyme deficiencies. It is a multisystemic disorder and can have highly varied presentations, often complicating patient identification.^1,2,4

Enzyme deficiencies may put patients at risk for progressive organ damage^1,2
Patients with a nonclassical phenotype may lack the overt skeletal manifestations that would lead to suspicion of MPS^3,4
It is important to know all the signs and symptoms of MPS, especially when patients do not present with classic signs (eg, bone deformity, short stature, and genu valgum)^3,4

Nonclassical musculoskeletal presentation can include^3,4:

Potential height of >140 cm
Joint stiffness and/or pain as the primary signs/symptoms
Lack of overt skeletal manifestations

Classical musculoskeletal presentation can include^1-3,6:

Height of <120 cm
Overt spinal and skeletal abnormalities
Joint laxity
Genu valgum
Abnormal gait
Chest abnormalities
Coarse facial features

MISSED DIAGNOSIS IS NOT UNCOMMON IN MPS

In a physician survey and systematic literature review, 44% (8/18) of patients with Morquio A had symptoms that raised concerns for or were misdiagnosed with spondyloepiphyseal dysplasia (SED) prior to testing for MPS.⁷

RECOGNIZE THE SIGNS

Morquio A is a multisystemic genetic disorder¹

Patients may present with different subsets of symptoms. Have you seen patients with:

Orthopedic Signs?^3,4

Bilateral Legg-Calvé-Perthes disease
Spondyloepiphyseal dysplasia
Multiple epiphyseal dysplasia
Pseudoachondroplasia
Undiagnosed skeletal dysplasia
Bilateral hip dysplasia
Vertebral body abnormalities: Hypoplasia, beaking, platyspondyly, kyphosis/gibbus
Odontoid dysplasia
Cervical spine instability

SEE HIP X-RAY ›

Ophthalmological Issues?^3,4,8

Diffuse corneal clouding
Cataracts
Reduction in visual acuity

SEE CLOUDY CORNEA ›

Cardiac Signs?⁹

Mitral and aortic valve stenosis and regurgitation
Tricuspid regurgitation
Hypertrophy

SEE IMAGE OF CARDIAC COMPLICATIONS ›

Pulmonary Complications?³

Obstructive sleep apnea
Respiratory infections

SEE X-RAY OF THE LUNGS ›

Multisystemic Signs?³

Reduced endurance
Loss of fine motor skills

Neurological Symptoms?^3,6

Cervical myelopathy
Cervical spine compression

Ear, Nose, and Throat Signs?^3,10,11

Conductive hearing loss
Sensorineural hearing loss
Otitis media
Enlarged tongue
Upper respiratory tract infections
Obstructed or restricted airways

SEE ENDOSCOPY OF AIRWAY ›

Gastrointestinal Signs?^3,10

Mild hepatosplenomegaly
Hernias
Diarrhea
Constipation
Abdominal pain

Rheumatological Signs?^3,12

Joint pain and stiffness
Joint laxity
Reduced hand function

SEE X-RAY OF THE HAND ›

Dental Issues?^3,13

Dentinogenesis imperfecta
Hypodontia
Pointed cusps
Spade-shaped incisors
Thin enamel
Abnormal buccal surfaces

SEE DENTAL IMAGE ›

MPS-SPECIFIC SURGICAL PLANNING

~75% of patients <12 years of age (n=172) and >95% of patients ≥12 years of age (n=153) in a multinational MPS IVA study required surgical or medical interventions.⁹

Life-threatening potential surgical complications for patients with MPS may include¹⁴:

Paralysis
Heart failure
Respiratory failure

High risk of anesthesia-related morbidity and mortality due to¹⁴:

Cervical instability and myelopathy
Cardiac abnormalities
Compromised airways and respiratory function

Surgical complications may account for mortality in 1 out of 10 patients with Morquio A (MPS IVA)¹⁵

Diagnosis of MPS is often delayed^5,16

MPS is a multisystemic and progressive disorder. Yet, the complex nature of skeletal dysplasias may delay diagnosis and lead to less optimal patient outcomes, including potential surgical risks.^1-3,15-18

Put patients on the path to disease‐specific management sooner by confirming or ruling out a diagnosis of MPS with no-charge testing from BioMarin.^7,17,18

FACILITATE DIAGNOSIS WITH A BROAD EVALUATION OF SKELETAL DYSPLASIAS^16-18

consider genetic testing with Discover Dysplasias™^16,18

Over 300 genes tested at no charge* for eligible patients¹⁸
Covers many of the most common skeletal dysplasias, including MPS^16,18
- Includes testing of genes for MPS I, II, III A-D, IV A/B, VI, and VII⁵
Board-certified genetic counselors are available to assist ordering physicians in reviewing results or counseling patients and their families if requested
Includes blood, saliva, and buccal swab sample collection options
Follow-up single-enzyme testing available for results indicative of any MPS
For eligible patients in the US and Canada. Please see DiscoverDysplasias.com for a full list of genes tested

Learn more at DiscoverDysplasias.com

Eligibility:

Discover Dysplasias^TM is available at no charge for eligible patients in the US and Canada suspected of having a skeletal dysplasia

*This is a sponsored testing program. While third parties and commercial organizations may provide financial support for this program, tests and services are performed by Invitae. Healthcare professionals must confirm that patients meet certain criteria to use the program. Third parties and commercial organizations may receive de-identified patient data from this program, but at no time would they receive patient identifiable information. Third parties and commercial organizations may receive contact information for healthcare professionals who use this program. Genetic testing and counseling is available in the US and Canada. Healthcare professionals and patients who participate in this program have no obligation to recommend, purchase, order, prescribe, promote, administer, use, or support any other products or services from Invitae or from third parties or commercial organizations.

UNCOVER RESOURCES THAT SUPPORT DIAGNOSIS AND CARE

Valuable information and tools are available to help from the start

BioMarin RareConnections™ provides product support services to help patients and caregivers access BioMarin therapies

BioMarin RareConnections™ provides a wide array of product support services to help patients and caregivers understand insurance access and coverage, provides information about financial assistance options, and coordinates delivery of BioMarin products for treatment.

CONNECT NOW ›

For Canadian Physicians ›

Connect With BioMarin for Diagnostic Support

Put patients on the path to disease-specific management sooner. Connect with BioMarin today for:

Local resources
One-on-one support throughout the diagnostic process
Information about receiving Discover Dysplasias™ testing kits for your eligible patients

CONNECT NOW WITH BIOMARIN ›

References:
1. Akyol MU, Alden TD, Amartino H, et al. Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance. Orphanet J Rare Dis. 2019;14(1):137. doi:10.1186/s13023-019-1074-9 2. Akyol MU, Alden TD, Amartino H, et al. Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance. Orphanet J Rare Dis. 2019;14(1):118. doi:10.1186/s13023-019-1080-y 3. Hendriksz CJ, Berger KI, Giugliani R, et al. International guidelines for the management and treatment of Morquio A syndrome. Am J Med Genet A. 2015;167A:11-25. doi:10.1002/ajmg.a.36833 4. Bhattacharya K, Balasubramaniam S, Choy YS, et al. Overcoming the barriers to diagnosis of Morquio A syndrome. Orphanet J Rare Dis. 2014;9:192. doi:10.1186/s13023-014-0192-7 5. Clarke L, Ellaway C, Foster HE, et al. Understanding the early presentation of mucopolysaccharidosis: results of a systematic literature review and physician survey. J Inborn Errors Metab Screen. 2018;6:1-12. doi:10.1177/2326409818800346 6. Tomatsu S, Montaño AM, Oikawa H, et al. Mucopolysaccharidosis type IVA (Morquio A disease): clinical review and current treatment. Curr Pharm Biotechnol. 2011;12:931-945. 7. Clarke L, Cristian I, Pollard L, et al. Simply Test for MPS™ Enzyme-Panel Program: a testing resource for early and accurate diagnosis of mucopolysaccharidoses. Poster presented at: ACMG Annual Clinical Genetics Meeting; April 10-14, 2018; Charlotte, NC. 8. Leslie T, Siddiqui MA, Aitken DA, et al. Morquio syndrome: electron microscopic findings. Br J Ophthalmol. 2005;89:925-926. 9. Harmatz P, Mengel KE, Giugliani R, et al. The Morquio A Clinical Assessment Program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects. Mol Genet Metab. 2013;109:54-61. 10. Hendriksz CJ, Al-Jawad M, Berger KI, et al. Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA. J Inherit Metab Dis. 2013;36:309-322. 11. Mesolella M, Cimmino M, Cantone E, et al. Management of otolaryngological manifestations in mucopolysaccharidoses: our experience. Acta Otorhinolaryngol Ital. 2013;33:267-272. 12. Lehman TJA, Miller N, Norquist B, et al. Diagnosis of the mucopolysaccharidoses. Rheumatology (Oxford). 2011;50(suppl 5):v41-48. doi:10.1093/rheumatology/ker390 13. Kinirons MJ, Nelson J. Dental findings in mucopolysaccharidosis type IV A (Morquio’s disease type A). Oral Surg Oral Med Oral Pathol. 1990;70:176-179. doi:10.1016/0030-4220(90)90114-8 14. Walker R, Belani KG, Braunlin EA, et al. Anaesthesia and airway management in mucopolysaccharidosis. J Inherit Metab Dis. 2013;36:211-219. 15. Lavery C, Hendriksz C. Mortality in patients with Morquio syndrome A. JIMD Rep. 2015;15:59-66. doi:10.1007/8904_2014_298 16. Nikkel SM. Skeletal dysplasias: what every bone health clinician needs to know. Curr Osteoporos Rep. 2017;15(5):419-424. doi:10.1007/s11914-017-0392-x 17. Jelin AC, O’Hare E, Blakemore K, et al. Skeletal dysplasias: growing therapy for growing bones. Front Pharmacol. 2017;8(79):1-6. doi:10.3389/fphar.2017.00079 18. Krakow D. Skeletal dysplasias. Clin Perinatol. 2015;42(2):301-319. doi:10.1016/j.clp.2015.03.003

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