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 …
FDA Approves First Gene Therapy for Treatment of Certain …
Jun 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 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 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 …
FDA Expands Approval of Gene Therapy for Patients with Duchenne ...
Jun 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 …
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” — …
FDA grants accelerated approval to first targeted treatment for …
The U.S. Food and Drug Administration today granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a …
FDA approves first gene therapy for Duchenne muscular dystrophy ...
Jun 23, 2023 · The FDA has granted accelerated approval to Sarepta’s gene therapy delandistrogene moxeparvovec (Elevidys) for Duchenne muscular dystrophy (DMD). It is the …
New Gene Therapy for Duchenne Muscular Dystrophy a …
Jun 26, 2023 · The new drug—delandistrogene moxeparvovec-rokl—is being developed by Sarepta Therapeutics and marketed under the name ELEVIDYS. We asked Ciafaloni to …
‘It’s a vote for hope’: first gene therapy for muscular dystrophy …
Jun 2, 2023 · Update: The US Food and Drug Administration has now approved the first gene therapy for Duchenne muscular dystrophy for children aged four and five. The one-time …