Understanding your child's genetic mutation

The Duchenne community is inspiring and supportive. There are many helpful resources and organizations to support families living with Duchenne. Here are just a few to get you started.

Sarepta does not endorse nor is responsible for the content of any of the listed websites or the services provided by these organizations. This is not a complete list of available websites.

  • Advocacy Groups and Nonprofit Organizations

    CureDuchenne
    A national nonprofit organization that raises awareness and funds to find a cure for Duchenne muscular dystrophy. The funds raised support the most promising research programs aimed at treating and curing the disease. CureDuchenne Cares provides outreach and education to parents and caregivers, as well as professional training and certification to physical therapists that treat Duchenne patients.

    Jett Foundation
    The mission of the Jett Foundation is to build awareness of Duchenne muscular dystrophy and fulfill the unmet needs in our community through programming, education, support, and research.

    Muscular Dystrophy Association (MDA)
    The MDA is leading the fight to free individuals - and the families who love them - from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. The MDA uses its collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases, caring for individuals from day one and empowering families with services and support in hometowns across America.

    Parent Project Muscular Dystrophy (PPMD)
    The largest and most comprehensive nonprofit organization in the United States focused on ending Duchenne. Its goal is to accelerate research efforts, catalyze advocacy, demand optimal care for all young men with Duchenne, and educate the global community.

  • Rare Disease Organizations

    EURORDIS
    A non-governmental patient-driven alliance of patient organizations representing 724 rare disease patient organizations in 64 countries.

    National Organization for Rare Disorders
    Provides a unified voice for the 30 million people who wake up every day to fight the battle with a rare disease, including parents and caregivers.

    Rare Disease Legislative Advocates
    A program of the EveryLife Foundation for Rare Diseases designed to support the advocacy of all rare disease patients and organizations.

    Genetic Alliance
    A nonprofit health advocacy organization that engages individuals, families, and communities to transform health. They create ways to make it easier to find or build solutions in health services and research.

    Global Genes
    A rare disease patient advocacy organization that aims to build awareness, educate the global community and provide critical connections and resources that equip advocates to become activists for their disease.

  • Patient Registries

    DuchenneConnect
    A comprehensive registry that connects the entire Duchenne community. This central hub brings together those living with the disease, along with their families, friends, and caregivers—and connects them with medical research, clinical care, clinical trials, and each other. Duchenne Connect is a program of Parent Project Muscular Dystrophy.

    TREAT-NMD
    A network for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Its focus is on the development of tools that industry, clinicians, and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide.

  • Clinical Trials

    ClinicalTrials.gov
    An online database of publicly and privately supported clinical studies conducted around the world. ClinicalTrials.gov currently lists thousands of studies with locations in all 50 states and in 191 countries.

  • Genetic Testing

    Genetics Home Reference
    An online resource from the National Institutes of Health. The website provides easy-to-understand information about genetic conditions and a range of topics. You'll find basic explanations of how genes work and how mutations cause disorders. It also includes current information about genetic testing, gene therapy, and the Human Genome Project.

    National Society of Genetic Counselors
    Provides a helpful, easy-to-use online directory to help connect physicians, patients, and other genetic counselors. Search by state, city, counselor's name, institution, work setting, type of specialty, or zip code.

    Decode Duchenne
    Provides free genetic testing, interpretation, and counseling to people with Duchenne or Becker muscular dystrophy who meet certain eligibility criteria and who have been unable to access genetic testing in the past due to financial barriers, such as cost or lack of insurance coverage. Decode Duchenne is administered by DuchenneConnect, a program of Parent Project Muscular Dystrophy.

  • Resources for Families and Caregivers

    ChildMuscleWeakness.org
    A website of the National Task Force for Early Identification of Childhood Neuromuscular Disorders. The site provides information and tools to help parents and healthcare professionals identify the early signs of muscle weakness and neuromuscular disease.

    Rare Share
    A unique social hub building communities for patients, families, and healthcare professionals affected by rare disorders.

    Caregiver Action Network (CAN)
    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, disease, or the frailties of old age. CAN (formerly the National Family Caregivers Association) is a nonprofit organization providing education, peer support, and resources to family caregivers across the United States free of charge.

    Center for Parent Information & Resources
    A central resource of information and products to the community of Parent Training Information Centers and the Community Parent Resource Centers so they can focus their efforts on serving families of children with disabilities.

    Cyberbullying Research Center
    Dedicated to providing up-to-date information about the nature, extent, causes, and consequences of cyberbullying, it is intended to be a resource for parents, educators, law enforcement officers, counselors, and others who work with youth. You will find facts, figures, and detailed stories from those who have been directly affected by online aggression. In addition, the site includes numerous resources to help you prevent and respond to cyberbullying incidents.

    Family Caregiver Alliance (FCA)
    A community-based nonprofit organization that that aims to illuminate the caregivers' daily challenges to better the lives of caregivers nationally, provide them the assistance they need, and champion their cause through education, services, research and advocacy. FCA offers programs at national, state, and local levels to support and sustain caregivers.

    Family Voices
    Family Voices aims to achieve family-centered care for all children and youth with special health care needs and/or disabilities. Throughout their national grassroots network, they provide families resources and support to make informed decisions, advocate for improved public and private policies, build partnerships among families and professionals, and serve as a trusted resource on health care.

    OurKids
    An email list of family of parents, caregivers and others who are working with children with physical and/or mental disabilities and delays.

    Parent to Parent USA
    Programs offering parent-to-parent support as a core resource for families with children who have a special health care need, disability, or mental health issue. Through a one-to-one “match,” experienced support parents provide emotional support to families and assist them in finding information and resources.

    Sibling Support Project
    A national program dedicated to the lifelong and ever-changing concerns of the millions of brothers and sisters of people with special health, developmental and mental health concerns.

    Stopbullying.gov
    A website providing information from various government agencies on what bullying is, what cyberbullying is, who is at risk, and how you can prevent and respond to bullying.

    The Bully Project
    Inspired by the award-winning film BULLY, this social action campaign includes tools for educators to spark meaningful conversations about bullying.

    The Parent Advocacy Center for Educational Rights (PACER Center)
    PACER Center is a parent training and information center for families of children and youth with all disabilities from birth to young adults. Parents can find publications, workshops, and other resources to help make decisions about education, vocational training, employment, and other services for their children with disabilities. PACER's National Bullying Prevention Center provides resources designed to benefit all students, including those with disabilities.

    The Washington State Fathers Network (WSFN)
    The Washington State Fathers Network (WSFN) connects men with other dads, resources, information and education, plus opportunities for “all family” celebrations. Focus is on assisting fathers as they become more competent and compassionate caregivers for their children with special needs. WSFN is the only regional program in the US and Canada fully devoted to fathers of children with special health care needs and developmental disabilities. Since 1986 it has opened new doors of hope for thousands of men and their families, providing them with support and resources to be effective, nurturing dads.

  • General Health Resources

    Agency for Healthcare & Research Quality (AHRQ)
    Aims to provide evidence to make health care safer, higher quality, more accessible, equitable and affordable, and to work with the US Department of Health and Human Services and with other partners to ensure that the evidence is used and understood.

    Health Hotlines (National Library of Medicine)
    A community service to help the public locate health-related information.

    Social Security Administration Compassionate Allowances Program
    Provides a way of quickly identifying diseases and other medical conditions that may qualify for financial assistance.

    Center on Technology & Disability
    Designed to increase the capacity of families and providers to advocate for, acquire, and implement effective assistive and instructional technology practices, devices, and services for those who suffer from disabilities.

    National Human Genome Research Institute
    Developed with the goal of mapping the human genome, the group provides a list of resources for financial assistance with genetic testing.

    National Library of Medicine
    Provides guidance on how to find reliable information online regarding human genetics.

    Patient Advocate Foundation
    Provides professional case management services to individuals facing barriers to healthcare access for chronic and disabling disease, medical debt crisis, and employment-related issues at no cost.

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