Innovative therapeutics


Patient Organizations

CLN2 Disease

Batten Disease Support and Research Association (BDSRA)
Founded in 1987 by parents seeking to build a network for those diagnosed with Batten disease, the BDSRA is the largest support and research organization dedicated to Batten disease in North America. It is committed to raising funds for research, providing family support services, enhancing education, raising awareness, and advocating for legislative action.

Noah’s Hope
Created by a family with 2 children affected by CLN2 disease, this organization has raised funds for research all over the world.

Lysosomal Storage Disorders

Lysosomal Diseases Australia
LDA is a national umbrella organization formed to represent the interests of people affected by lysosomal storage disorders including their families and care givers, and to maximize resources available for support services, treatment, and research.

Lysosomal Diseases New Zealand (LDNZ)
LDNZ is committed to improving contacts, information sharing and support among affected people and their families, within New Zealand and internationally; to advocate for and support accelerated research into the causes and treatment of Lysosomal Storage diseases; to advocate for and support improvements to the clinical care of affected people.

Vaincre les Maladies Lysosomales-France (VML)

 Mucopolysaccharidosis (MPS)

MPS I Resource Center

This website was created to provide information regarding MPS I to patients, families, caregivers, and healthcare professionals. The site contains information about the disease, support programs and online resources to manage the challenges associated with MPS I.

MPS VI Resource Center

This website was created to provide information regarding MPS VI to patients, families, caregivers, and healthcare professionals. The site contains information about the disease, support programs and online resources to manage the challenges associated with MPS VI.

Morquio Parent Support Group, International Morquio Organization

The International Morquio Organization is dedicated to seeking out people who have Morquio Type A in order to provide a mutual aid network; act as a advocate between patients, physicians and scientists; compile medical information into a database; and pursue funding for education, families, and research. The site provides an MPS IVA patient registry and links to other support and advocacy groups.

International MPS Societies

 Phenylketonuria (PKU)

National PKU Alliance

The NPKUA is the national patient voice in the PKU community and has proven itself to be thoughtfully aggressive in helping families who need it most. The NPKUA’s four signature programs include: research, education, support and advocacy.

Children’s PKU Network (CPN)

CPN is dedicated to raising public awareness, educating and providing direct assistance through a variety of programs, and to helping people with PKU and other metabolic disorders reach their full potential.

Canadian PKU and Allied Disorders Inc. (CanPKU)

CanPKU is the Canadian non-profit organization dedicated to providing up-to-date, accurate news and information to families and professionals dealing with PKU and allied disorders. Our focus is on advocating for better care and coverage of treatments, and hosting education events… Because Knowledge Leads to Better Health.

Genetic/Rare Diseases

The Genetic Alliance

The Genetic Alliance is dedicated to helping individuals and families with genetic disorders. The site provides information about a broad spectrum of genetic diseases and links to numerous resource organizations.

Children Living With Inherited Metabolic Disease (CLIMB)

CLIMB is committed to fighting metabolic diseases through research, awareness, and support.  The organization is dedicated to providing advice, information, and support on all metabolic diseases to children, young adults, families, caregivers, and professionals.


MUMS is a national parent-to-parent organization for parents or care providers of a child with any disability, rare, or not so rare disorder, chromosomal abnormality or health condition. MUMS provides support to parents in the form of a networking system that matches them with other parents whose children have the same or similar condition.

National Organization for Rare Disorders (NORD)

NORD is the federation of voluntary health organizations dedicated to helping people with rare “orphan” diseases and assisting the organizations that serve them. The site provides searchable indexes and databases of rare diseases and organizations, and a medication assistance program as well as up-to- date news and information.

Global Genes​

Global Genes​ is a non-profit patient advocacy organization working to eliminate the challenges of​ rare disease by building awareness, educating the global community, and providing critical connections and resources that equip advocates to become activists for their disease. Numerous tools, resources and educational events can be found on the Global Genes website.

Additional Resources


PUBMED is the U.S. National Library of Medicine’s free access to the MEDLINE database. The site contains a vast collection of published medical information from scientific and medical journals.

The National Institutes of Health (NIH)

The NIH is the premiere medical research organization for the United States . This site provides information about scientific research, drug therapies, and clinical trials.

Note: These listings are provided by BioMarin as additional information for patients, their families and their healthcare providers. The web pages and their content are maintained by the organizations listed above. With the exception of its own websites, BioMarin does not endorse any particular organization or the content contained on their website.

Read our patient stories…



Kendra - BioMarin Patient Stories

Watch our patient videos…

Kendra - video

Annabelle - video



Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU



Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361

Global Medical Information

Contact Global Medical Information