A service of the U.S. National Institutes of Health that serves as a registry and database containing information about publicly and privately-funded human clinical studies being conducted around the world. Some studies are pending, and still recruiting, while others are ongoing.
Specific entry detailing the present Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics (exactly how the drug behaves in the human body) of Pfizer’s investigational drug, PF-06252616, in Duchenne Muscular Dystrophy. Provides professionals detailed information about the purpose, design, and outcome measures of the proposed clinical trial.
A service of the U.S. National Library of Medicine/National Institutes of Health that explains what clinical trials entail; their purpose, design, oversight, and other details regarding the general definition of a clinical trial.
National Heart Lung and Blood Institute
A service of the U.S. Department of Health and Human Services/National Institutes of Health/National Heart, Lung and Blood Institute, that consists of a documentary video for parents about enrolling their children in clinical trials.
National Human Genome Research Institute
A service of the National Institutes of Health/National Human Genome Institute (Advancing human health through genomics research) that provides detailed information about Duchenne Muscular Dystrophy. This website includes information for patients and their parents about every aspect of the disease, including ongoing clinical research projects, and a comprehensive list of additional informational resources.
Duchenne Foundation Australia
A member of the United Parents Projects, Duchenne Foundation is dedicated to “using national and international collaboration to improve the quality and quantity of life for those living with Duchenne and Becker muscular dystrophy and their families, through research, education and advocacy.” Focused on facilitating Australian research into these neuromuscular diseases, and providing support for patients and their families.
Muscular Dystrophy Canada
Muscular Dystrophy Canada is a national, non-profit organization of dedicated volunteers and staff who work to provide support and resources to clients impacted by 1 of over 150 neuromuscular disorders. Muscular Dystrophy Canada’s mission is to enhance the lives of those affected with neuromuscular disorders by continually working to provide ongoing support and resources while relentlessly searching for a cure through well funded research.
DMD China Care and Support Association
Founded in 2011 by parents of boys with DMD/BMD in Beijing, China. Focused on helping the estimated 100,000 boys in China with one of these diseases, the organization brings together clinicians and researchers, providing updates on international research information. Also maintains communication links with international community groups and local patients. Content in Chinese (Mandarin dialect)
Parent Project is a non-profit organization run by parents of children diagnosed with Duchenne (DMD) and Becker (BMD) Muscular Dystrophy. It was founded in the USA in 1994 and in 2001, an organization was started in the Czech Republic. Parent Project Muscular Dystrophy mission is to improve the treatment, quality of life and long-term outlook for all individuals affected by Duchenne Muscular Dystrophy (DMD) through research, advocacy, education and compassion. Content in Czech, German, or English.
Association Française Contre les Myopathies-AFM
A French organization that combines the efforts of patients and their families to combat genetic diseases specifically, and rare diseases in general. Dedicated to fundraising and “scientific, medical, technological, and social” innovations to accelerate research, enhance communication about neuromuscular disease research, and find cures for diseases such as DMD. Content in French.
Duchenne Parent Project France & Monaco
Duchenne Parent Project France is the non-profit association local branch of a European and International network of organizations all founded and managed by parents of Duchenne children (USA, UK, Australia, Netherlands, Germany, Italy, Monaco). With Duchenne Parent Project France, parents are fighting against this progressive and deadly disease affecting their boys.
Aktion Benni & Co/Parent Project
An organization dedicated to patients and their families dealing with DMD. Based in Germany. Content in German.
Fundation Gyógyító Jószándék
A foundation for families caring for DMD children. Content in Hungarian.
The foundation aims to provide a networking platform for the DMD community and increase the chance of eventually finding a cure for the children affected by the disease. The Misko Foundation keeps track of research conducted in Hungary and abroad and publishes summaries of these efforts. The foundation also plans to finance DMD research and provide researchers and doctors with grants to finance their studies and professional training.
Duchene Ireland is a patient organization and registered Irish Charity (CHY 18197) established to facilitate funding translational research into Duchenne Muscular Dystrophy. Affiliated with numerous patient groups and clinical, research, and support networks for DMD, Duchenne Ireland works internationally to rapidly advance research into Duchene and welcomes organizations, parents, and supporter who believe they can help advance progress in this research. Content in English.
Little Steps Association
Little Steps is an organization founded in Israel by parents of children with Duchenne and Becker muscular dystrophy diseases. Little Steps seeks to be a second “home” for the children and their families who are coping with physical and mental difficulties caused by the disease. Content in Hebrew. Some content in English.
UPA! Cura Duchenne
Leslie Guzman established UPA! Cura Duchenne in 2007 with the purpose of integrating under one emblem and name the diverse efforts of leading Latin and Ibero-American organizations focused on treatment, therapies, and a cure for Duchenne and other neuromuscular disorders. Content in Spanish.
Duchenne Parent Project
Guided by foundation president Elizabeth Vroom, the Duchenne Parent Project is an organization founded by parents of children with Duchenne muscular dystrophy. The project’s website contains information about DMD and the foundation itself, including activities and Duchenne Heroes. Content in Dutch.
United Parent Project Muscular Dystrophy
United Parent Projects Muscular Dystrophy (UPPMD) is owned and managed by parent project organizations set up by parents of children with Duchenne Muscular Dystrophy in many different countries all over the world. These national projects are run by parents for parents. Content in English.
Muscular Dystrophy Association of New Zealand
An organization aimed to provide personal support and information to New Zealanders living with neuromuscular conditions. It also seeks to advocate, influence and promote equality of opportunity. Its vision is that people living with neuromuscular conditions have unrestricted opportunities to achieve their full potential.
InsamlingsStiftelsen för Muskel DystrofiForsknings
The Foundation for Muscular Dystrophy Research was founded in 1986 by Elwyn and Christina Mandley and Jarl Mared. Its purpose is to support research and disseminate information on Duchenne and Becker muscular dystrophy. Since its beginning, the foundation has supported several research projects and organized several symposia. The foundation is supported entirely by private contributions and the board and executive members work as volunteers. Content in Swedish.
Progena was founded in 2006 by the parents of a Duchenne boy who didn’t find much help from existing infrastructures when they had to come to terms with their son’s diagnosis. The Progena Foundation organizes family outings and other events. Content in French.
A British website/organization dedicated to funding research to find a cure or viable treatments for Duchenne muscular dystrophy. Also runs education and transition projects for young people living with DMD.
The mission of Alex’s Wish is to bring about new effective treatments to help delay the onset of symptoms in boys with Duchenne. The organization hopes to eradicate Duchenne for all in the next 5-10 years. It has invested and pledged £130k to promising new treatments and is currently reviewing the best places to put the remaining funds raised to date.
Duchenne Children’s Trust
The Duchenne Children’s Trust was set up by Emily and Nick Crossley, after their son, Eli was diagnosed with Duchenne Muscular Dystrophy. Its mission is to raise money to give to the best global research effort – to fund a treatment or cure in time to save Eli’s life and the hundreds of thousands of other boys like him.
Duchenne Family Support Group
This mission of this organization is to provide support for families. DFSG was started in 1987 by a small group of parents with children diagnosed with DMD. DFSG is “a national charity run by families for families affected by DMD. It provides a positive national support network of parents, their families and professionals. Located in London, England.
Duchenne Now is dedicated to finding and funding treatments and eventual cure for ALL living with Duchenne and Becker MD. Duchenne Now was officially launched on March 1st 2012, with a fresh approach to achieving our goals.
Harrison’s Fund’s one goal is to get as much money as possible into the hands of the world’s best researchers, who are working to find a cure for Duchenne. It differentiates itself from many other Duchenne charities by focusing on treatment rather than palliative care.
Joining Jack is an organization determined to continue to do all it can to raise awareness, money and ultimately to find a cure for Duchenne Muscular Dystrophy to help Jack, and the thousands of other boys affected by DMD. Content in English.
Muscular Dystrophy UK
Previously known as the Muscular Dystrophy Campaign, Muscular Dystrophy UK is a charity bringing individuals, families and professionals together to beat muscle-wasting conditions. Founded in 1959, it’s been leading the fight against muscle-wasting conditions since then. It brings together more than 60 rare and very rare progressive muscle-weakening and wasting conditions, affecting around 70,000 children and adults in the UK. Content in English.
Treat-NMD/Neuromuscular Network is a UK-based organization dedicated to ensuring that the most promising new treatments for neuromuscular disorders reach patients as quickly as possible. Find links to numerous organizations around the globe – each dedicated to providing support for patients and their families dealing with Duchenne muscular dystrophy (DMD) and Becker Muscular dystrophy (BMD). Specific organization descriptions and links follow:
This U.S.-based organization was founded to funnel financial resources towards finding a cure. Other organizations are focused on providing funds to assist families struggling to care for a child with DMD. According to Charley’s Fund’s mission statement: “…Our sole mission is to fund a cure or treatment for Duchenne. We invest our money in translational research – research that focuses on moving science from the lab into human clinical trials.”
The mission of Coalition Duchenne is to raise global awareness for Duchenne muscular dystrophy, to fund research and to find a cure for Duchenne.
Founded by parents whose only son has DMD, CureDuchenne is described as a grassroots organization dedicated to “moving research into practice.” According to the non-profit organization’s website: “CureDuchenne’s accelerated push to move research from the lab into clinical trials could halt the progression of the disease and save the lives of thousands of Duchenne boys.”
Duchenne Connect is a central hub at Emory University that links the resources and needs of those living with Duchenne/Becker muscular dystrophy to the professional community, including clinicians, policymakers, industry professionals, and medical researchers.
Duchenne Muscular Dystrophy Research Fund
DMDfund is a non-profit organization founded by parents, relatives, and friends of kids who have DMD. DMDfund is dedicated to finding a cure for DMD by funding research, so that millions of kids worldwide can live. DMDfund is a distinct and independent organization, and is not affiliated with, nor receives any funding from, any other organization.
Duchenne San Diego
Duchenne San Diego is San Diego’s only non-profit organization whose exclusive purpose is to raise funds for Duchenne Muscular Dystrophy (DMD). Its hope is to make a positive impact on San Diego’s community of individuals and families who live with Duchenne by enhancing their quality of life.
Foundation to Eradicate Duchenne
A 501c(3) nonprofit organization established in 2002 with the goal of finding treatments and an ultimate cure for Duchenne Muscular Dystrophy. This organization is established by Dana and Joel Wood of Alexandria, Virginia whose son James Wood was diagnosed in May 2000 with Duchenne Muscular Dystrophy. They have raised nearly $10 million in private donations and worked with Congress to secure nearly $40 million in federal appropriations since James was diagnosed.
Hope for Gus
The mission of Hope for Gus is to raise research dollars and awareness of Duchenne Muscular Dystrophy. It is committed to funding research that will result in effective treatments in the near-term. Its specific focus is on treatments that will preserve muscles in DMD boys while researchers continue to search for the cure.
Living each day as it comes. Enjoying each and every moment. Helping Liam and other boys with DMD. Experience life to its fullest. Preparing for the future. A nonprofit for dedicated to supporting families, furthering research, and raising awareness about DMD.
Muscular Dystrophy Association
MDA is the world’s leading nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education.
Parent Project Muscular Dystrophy (USA)
The largest nonprofit in the U.S. focused exclusively on Duchenne MD. Parent Project Muscular Dystrophy’s mission is to end Duchenne. It accelerates research, raises voices in Washington, demands optimal care for all young men, and educates the global community.
The mission of Ryan’s Quest is to increase awareness of Duchenne muscular dystrophy with the purpose of allocating funds for research that has the greatest potential of finding a cure or treatment for this disease. Ryan’s Quest is primarily focused on supporting research for near-term, potential therapeutics and better testing platforms (tools) for development of DMD compounds. It is also interested in helping support families that are enrolled in clinical trials.
Established in 2002, Suneel’s Light is a charitable foundation whose namesake, Suneel Ram, is a boy who suffers from Duchenne Muscular Dystrophy. The foundation was established to fund critical research for treatments and a cure for Duchenne Muscular Dystrophy.
The Jett Foundation
The mission of the Jett Foundation is to increase worldwide awareness of Duchenne Muscular Dystrophy with the purpose of raising and appropriating funds for research that will realize a cure for this fatal disease and ensure that children like Jett have the opportunity to enjoy a full life. The Jett Foundation sponsors week-long camps for children with DMD.