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...

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...

DMD Muracchini started as a small kiosk store on the 10th floor of a building on Jeppe Street, and then moved into their first boutique store when the Carlton Centre was built. Currently we have 6 exclusive DMD stores where you can find the original DMD styled clothing, footwear and accessories that you love to love.

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.

3 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...

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.

2025年3月18日 · In the phase 3 Embark trial that won Elevidys its broad label that covers all DMD patients ages 4 and older, the Sarepta med was found not significantly better than placebo on a measure of motor ...

2024年2月16日 · Elevidys, which was granted approval in June 2023, is currently indicated to treat ambulatory pediatric DMD patients between the ages of four and five years with a confirmed mutation in the DMD gene. The single-dose gene transfer therapy uses a recombinant adeno-associated virus vector serotype 74 (AAV74) to deliver a microdystrophin transgene ...

6 天之前 · DMD is caused by a mutation in the DMD gene that disrupts the production of dystrophin, a protein that provides structural support and protection to muscles. ... Because of these known side effects, Elevidys prescribing label notes that patients with a preexisting liver disease, either chronic or acute — hepatic viral infection is named as an ...

22 小时之前 · Subjects may have the opportunity to join an open-label extension study, where the long-term efficacy, tolerability, and safety of the therapy will be assessed. ... had lifted a clinical hold on DMD therapy, ENTR-601-44, and authorised the commencement of ELEVATE-44-102, a Phase Ib MAD clinical study for the therapy. Sign up for our daily news ...