Advocacy and Resources
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Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophy (PPMD) fights to end Duchenne. They accelerate research raise their voices to impact policy, demand optimal are for every single family, and strive to ensure access to approved therapies.
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Foundation for Angelman Syndrome Therapeutics (FAST)
FAST is the leading patient advocacy organization working to cure Angelman syndrome. As the largest non-governmental funder of Angelman syndrome research in the world, our goal is to drive forward transformative research and development programs as quickly as possible for those living with Angelman syndrome — regardless of age or genotype.
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CureDuchenne
CureDuchenne is recognized as the global leader in research, patient care and innovation for improving and extending the lives of those with Duchenne.
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Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA's mission is to empower the people we serve to live longer, more independent lives.
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National Organization for Rare Disorders (NORD)
NORD advances practical, meaningful, and enduring change so people with rare diseases can live their fullest and best lives. Every day, we elevate care, advance research, and drive policy in a purposeful and holistic manner to lift up the rare disease community.
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Cure SMA
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease that affects the motor nerve cells in the spinal cord and impacts the muscles used for activities such as breathing, eating, crawling, and walking.
Cure SMA leads the way to a world where everyone impacted by spinal muscular atrophy is empowered to lead independent, successful, and fulfilling lives.
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Friedreich's Ataxia Research Alliance (FARA)
FARA's Mission is to marshal and focus the resources and relationships needed to cure FA by raising funds for research, promoting public awareness, and aligning scientists, patients, clinicians, government agencies, pharmaceutical companies and other organizations dedicated to curing FA and related diseases.
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Emily's Entourage
Emily’s Entourage speeds lifesaving research and drug development for individuals in the final 10% of the cystic fibrosis (CF) community that do not benefit from currently available mutation-targeted therapies, including those with nonsense mutations of CF.
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Facioscapoulohumeral Muscular Dystrophy (FSHD) Society
The FSHD Society is the world’s largest research-focused patient organization for facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy. We have catalyzed major advancements and are accelerating the development of treatments and a cure to end the pain, disability, and suffering endured by one million people worldwide who live with FSHD.
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Prader-Willi Syndrome Association
Prader‐Willi Syndrome Association | USA was formed in 1975 to unite parents, professionals, and other interested citizens to enhance the quality of life of those affected by Prader‐Willi syndrome. PWSA | USA empowers the PWS community through shared experiences, research, education, advocacy, and support. With chapters in most states, ours is the only national PWS support organization whose sole purpose is to assist individuals with the syndrome, and their families, every step of the way.
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Cure VCP Disease, Inc.
Cure VCP Disease, Inc., is dedicated to driving the development of a cure for diseases caused by the valosin containing protein (VCP) gene, including muscle disease, bone disease, and the neurodegenerative diseases ALS, FTD, Parkinson’s, and CMT.
We collaborate on research and advocacy initiatives that help patients and their families. We encourage patients and doctors to connect with us.
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Jett Foundation
Jett Foundation empowers people and families impacted by Duchenne muscular dystrophy through the development of transformative programming, educational opportunities, and ongoing support for every stage of a Duchenne journey.
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Global Genes
The mission at Global Genes is to provide patient advocates with a continuum of services to accelerate their path from early support and awareness through research readiness, using a collaborative approach that involves biopharma, researchers and funders, with data as a central core.
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We empower the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments, and cures.
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Caregiver Action Network
Caregiver Action Network (CAN) is the nation’s leading family caregiver organization working to improve the quality of life for the more than 90 million Americans who care for loved ones with chronic conditions, disabilities, or disease, as well as those supporting the living needs of older adults. CAN serves a broad spectrum of family caregivers ranging from the parents of children with significant health needs, to the families and friends of wounded soldiers; from a young couple dealing with a diagnosis of MS, to adult children caring for parents with Alzheimer’s disease.
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Genetic and Rare Disease Information Center (GARD)
The mission of the Genetic and Rare Diseases (GARD) Information Center is to help the rare disease community meet these common challenges. We do this by providing free access to reliable and easy-to-understand information. We support the rare disease community both through our website and through the dedicated Information Specialists in our Contact Center.