The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has once again snubbed Translarna ...

CHMP upheld earlier opinions that Translarna is not of significant benefit to DMD due to nonsense mutations; European ...

Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead ...

A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular ...

In a study published in the journal Nature, researchers from the Center for Genomic Regulation (CRG) and the Wellcome Sanger Institute have discovered that mutations affect protein stability ...

EMA re-confirms non-renewal of conditional marketing authorisation of Duchenne muscular dystrophy medicine, Translarna: Amsterdam, The Netherlands Monday, October 21, 2024, 10:00 ...

Protein behavior can be predicted with simple math Mutations affect protein stability following remarkably simple rules Date: September 25, 2024 Source: Center for Genomic Regulation Summary ...

From this exon skipping approach, the company has generated three commercial assets treating DMD, a monogenic, X-linked disease caused by mutations that encode for the dystrophin protein.

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has maintained its negative opinion on the renewal ...