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EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This...
ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age: • For patients who are ambulatory and have a confirmed mutation in the DMD...
FDA broadens label for Sarepta's Duchenne gene therapy Elevidys
2024年6月21日 · On Thursday, the FDA expanded the label for Elevidys (delandistrogene moxeparvovec-rokl) to all DMD patients ages 4 and older. It’s a major boost for the first gene therapy to treat the inherited...
Sarepta Wins Full Approval and Label Expansion for DMD Gene …
2024年6月20日 · Sarepta Therapeutics’ gene therapy Elevidys will be available to Duchenne muscular dystrophy (DMD) patients who are at least 4 years old, regardless of whether they can walk, the FDA said Thursday. This expands the former accelerated approval for the gene therapy in four and five year olds who were still ambulatory.
FDA Approves Nonsteroidal Treatment for Duchenne Muscular …
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...
Sarepta bags key expansion to DMD gene therapy label in US
6 天之前 · Elevidys (delandistrogene moxeparvovec) can now be used to treat both ambulatory and non-ambulatory boys aged four and above with DMD, a major expansion on its earlier accelerated approval with a...
Label for DMD gene therapy Elevidys expanded in the US
2024年6月21日 · Elevidys (also known as delandistrogene moxeparvovec, or dela mox, and SRP-9001) is an adeno-associated virus (AAV) vector-based gene therapy product for ambulatory patients with a confirmed mutation in the DMD gene.
Sarepta Obtains Broader DMD Label For Elevidys - Scrip
Sarepta Therapeutics, Inc. obtained expanded US Food and Drug Administration approval of its Duchenne muscular dystrophy gene therapy Elevidys in ambulatory boys aged 4 and older, overcoming concerns after a confirmatory trial missed its primary endpoint last October.
AMONDYS 45 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. This...
NS Pharma's Duchenne drug Viltepso flunks confirmatory trial
2024年5月28日 · In August of 2020, the FDA granted Viltepso accelerated approval for patients with DMD amenable with exon 53 skipping. The nod was based in part on the results of a phase 2, open-label,...