Sarepta Simultaneously Pursues Exon Skipping, PPMO, and Gene Therapies for DMD
Sarepta Therapeutics will be seeking FDA approval for its investigational gene therapy, SRP-9001, to treat Duchenne muscular therapy.
DMD Features
Gene Therapy Could Emerge as a DMD Treatment in 2023
Michael Kelly, MD, chief scientific officer at CureDuchenne, says a gene therapy for DMD could win FDA approval as early as 2023.
A Unique Way to Raise Money for DMD Research: Cattle Auctions
Montana cattle ranchers Paul and Laura Heaton hold an annual livestock auction to raise money to help their son, Grant, who has DMD.
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The Alliance for Regenerative Medicine Fights for Better Cell, Gene Therapies
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
Female Carriers of Duchenne Urged to Seek Early Treatment for Heart Problems
Pradeep Mammen, MD, an expert on DMD-linked cardiomyopathy, said female carriers of Duchenne face a significant risk of heart failure later in life.
Patient Advocates Seek to Raise Awareness of DMD Among Spanish Speakers
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.
Nonprofit Group Team Joseph Helps Meet the Needs of Patients With DMD
The nonprofit Team Joseph raises money for scientific studies, but also looks for immediate gaps or needs not being met by other patient organizations.
Businessman Driven to Cure DMD After a Devastating Family Diagnosis
Ilan Ganot, CEO of Solid Biosciences, cofounded the company with his wife, Annie, after their son, Eytani, was diagnosed with Duchenne in 2012.
Duchenne Gene Therapy Pioneer Dr. Jerry Mendell Reflects on a Lifetime of Research
Jerry R. Mendell, MD, professor of neurology and pediatrics at Nationwide Children’s Hospital has devoted his life’s work to curing DMD.
Poster Child for ‘Right to Try’ Movement Marks 5 Years on Novel DMD Treatment
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.
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