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 ...

Recently presented data demonstrated sustained functional improvement with DYNE-251 treatment through 18 months - - Data from the fully enrolled DELIVER registrational expansion cohort is planned for ...

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD ... has three FDA-approved drugs that use "exon skipping" technology. These allow the ...

Italfarmaco’s oral HDAC inhibitor givinostat has been approved in the US as a treatment for Duchenne muscular dystrophy (DMD ... Sarepta and NS Pharma’s exon-skipping therapies, which ...

U.K. regulators cleared a multiple myeloma drug GSK withdrew from market three years ago. Elsewhere, an investment firm pressed Elevation Oncology to liquidate and the FDA relaxed risk monitoring ...

Shares of Dyne Therapeutics (NASDAQ: DYN) climbed 3.9% following the announcement that the European Commission has granted orphan drug designation to the companys ...

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Chiefly focused on Duchenne, the Duchenne Muscular Dystrophy Research Program (DMDRP) considers “a broad range of unanswered research questions that are potentially critical to treating Duchenne ...