FDA expands approval of first gene therapy for rare form of muscular …
Jun 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...
FDA approves $3.2 million gene therapy for rare muscular dystrophy …
Jun 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.
New Gene Therapy for Duchenne Muscular Dystrophy
Oct 23, 2024 · Without dystrophin, muscle becomes fragile and easily degenerates. A new single-dose intravenous gene therapy, delandistrogene moxeparvovec, creates “micro-dystrophin” — a scaled-down version of the large gene that encodes dystrophin — …
New drugs bring hope and confusion for Duchenne families - STAT
Sep 9, 2024 · Ultimately, what patients with Duchenne deserve is a treatment that restores full-length dystrophin, and preferably one that can revert muscle damage, to truly improve long-term outcomes.
Muscular dystrophy patients get first gene therapy - NPR
Jun 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 advisers narrowly vote in favor of experimental gene therapy …
May 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 Expands Approval of Gene Therapy for Patients with Duchenne ...
Jun 20, 2024 · The FDA expanded the approval of Elevidys, a gene therapy for the treatment of Duchenne muscular dystrophy for ambulatory and non-ambulatory individuals 4 years of age and older with DMD with a...
FDA Approves First Gene Therapy for Treatment of Certain …
Jun 23, 2023 · 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 a defective...
New Gene Therapy for Duchenne Muscular Dystrophy a …
Jun 26, 2023 · Last week, the U.S. Food and Drug Administration (FDA) granted accelerated approval of a new drug to treat ambulatory pediatric patients aged 4-5 years with Duchenne muscular dystrophy (DMD).
FDA grants accelerated approval to first targeted treatment for …
Today, the FDA approved a new treatment for Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients.