Italfarmaco's oral HDAC inhibitor Duvyzat has been recommended for approval in the EU as a treatment for Duchenne muscular ...

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The EC has granted orphan drug designation to Dyne Therapeutics’ DYNE-251 for treating Duchenne muscular dystrophy (DMD).

This year's Futures National Conference, hosted by CureDuchenne for the Duchenne and Becker MD communities, is scheduled to ...

Detailed price information for Dyne Therapeutics Inc (DYN-Q) from The Globe and Mail including charting and trades.

It's been nearly two years since Elevidys, a gene therapy for Duchenne MD developed by Sarepta Therapeutics, was approved by ...

First, there might be a specific region within the dystrophin gene that is altered, creating either a dysfunctional protein or a truncated protein. This dystrophin transcript, theoretically ...

It offers EXONDYS 51 injection to treat Duchenne muscular dystrophy (Duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the ...

However, postoperative immunohistochemical examination confirmed the diagnosis of BA. Next-generation sequencing (NGS) further revealed an EGFR exon 19 deletion mutation. Conclusion: The histological ...

The findings suggest that the body's own immune system may be undermining the treatment with gene therapy such as Elevidys (delandistrogene moxeparvovec) aimed at reintroducing the missing protein ...

“Drugs that induce exon skipping are intended to convert out-of-frame mutations ... led to the development of new gene therapies that use viral vectors to re-express dystrophin. This strategy, Górecki ...

Duchenne muscular dystrophy is caused by the absence of the protein dystrophin. Therapies for DMD, including Elevidys, work to reintroduce dystrophin — but the immune system might perceive ...