FDA expands approval of first gene therapy for rare form of muscular …
WEBJun 20, 2024 · Duchenne muscular dystrophy causes progressive muscle weakness that can rob children of their ability to walk by the time they’re teenagers, and many don’t live well into their 30s. It...
Muscular dystrophy patients get first gene therapy - NPR
WEBJun 22, 2023 · The Food and Drug Administration approved the first gene therapy for Duchenne muscular dystrophy, but limited access to those ages four and five.
FDA approves $3.2 million gene therapy for rare muscular dystrophy …
WEBJun 22, 2023 · Duchenne muscular dystrophy causes progressive muscle weakness that can rob kids of their ability to walk by the time they’re teenagers, and many don’t live well into their 30s. It affects an...
FDA Expands Approval of Gene Therapy for Patients with Duchenne ...
WEBJun 20, 2024 · Duchenne muscular dystrophy is a rare and serious genetic condition which worsens over time, leading to weakness and wasting away of the body’s muscles. The disease occurs due to...
FDA Approves First Gene Therapy for Treatment of Certain …
WEBJun 23, 2023 · Today, the U.S. Food and Drug Administration approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD) with...
FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy
WEBMar 21, 2024 · Today, the U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older. Duvyzat...
New Gene Therapy for Duchenne Muscular Dystrophy a …
WEBJun 26, 2023 · The new drug—delandistrogene moxeparvovec-rokl—is being developed by Sarepta Therapeutics and marketed under the name ELEVIDYS. We asked Ciafaloni to describe the drug, why the FDA chose to limit its use, …
MDA 2024: Gene therapy’s benefits most pronounced in young boys
WEBMar 7, 2024 · Among ambulatory boys with Duchenne muscular dystrophy (DMD), a one-time treatment with investigational gene therapy fordadistrogene movaparvovec has helped preserve functions and increase muscle volume for three years, especially in the youngest patients.
FDA advisers narrowly vote in favor of experimental gene therapy …
WEBMay 12, 2023 · SRP-9001, an experimental gene therapy that aims to slow or stop the progression of Duchenne muscular dystrophy, is being considered for accelerated approval by the FDA.
FDA Approves Duvystat, New Oral Treatment for Duchenne Muscular ...
WEBMar 28, 2024 · A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. Boys 6 years and older can take Duvystat, to slow the course of the illness.