Sarepta Therapeutics will be seeking FDA approval for its investigational gene therapy, SRP-9001, to treat Duchenne muscular therapy.
Michael Kelly, MD, chief scientific officer at CureDuchenne, says a gene therapy for DMD could win FDA approval as early as 2023.
Montana cattle ranchers Paul and Laura Heaton hold an annual livestock auction to raise money to help their son, Grant, who has DMD.
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
Pradeep Mammen, MD, an expert on DMD-linked cardiomyopathy, said female carriers of Duchenne face a significant risk of heart failure later in life.
Thousands of families affected by Duchenne in the US and Latin America face the extra hurdle of accessing reliable information on the disorder in Spanish.
The nonprofit Team Joseph raises money for scientific studies, but also looks for immediate gaps or needs not being met by other patient organizations.
Ilan Ganot, CEO of Solid Biosciences, cofounded the company with his wife, Annie, after their son, Eytani, was diagnosed with Duchenne in 2012.
Jerry R. Mendell, MD, professor of neurology and pediatrics at Nationwide Children’s Hospital has devoted his life’s work to curing DMD.
A video of Jordan McLinn, who has DMD, went viral when he tried to embrace then-President Trump before he signed the “Right to Try” bill at the White House.