Treatments

Sarepta Therapeutics, Inc., a leader in precision genetic medicine for rare diseases, announced positive results from its interim analysis of muscle biopsy endpoints comparing casimersen [...]

February 25, 2019 Santhera Pharmaceuticals announces results from the SYROS study.   The primary objective of this study was to evaluate the long-term evolution of the respiratory function [...]

Sarepta Announces FDA Acceptance of Golodirsen (SRP-4053) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53 Press release here: Sarepta.com   [...]

Here’s the latest news from PTC Therapeutics about Translarna™ (ataluren). In a recent press release, PTC announces that Translarna™ (ataluren) slows disease progression in children with Duchenne [...]

Here’s the latest news from PTC Therapeutics about Deflazacort (EMFLAZA®). A publication of data in Muscle & Nerve comparing the efficacy and safety of deflazacort and [...]

  Here’s the latest news from Sarepta Therapeutics press release about EXONDYS® (eteplirsen). EXONDYS® (eteplirsen) is designed to treat patients with Duchenne muscular dystrophy (DMD) [...]

  One of the objectives of our team is to inform you about new treatments. Here’s the latest news from PTC Therapeutics press release about Translarna ™ (ataluren). Enjoy reading and [...]

  Sarepta Therapeutics is recruiting DMD patients that have out-of-frame deletion mutations in dystrophin that may be treated by skipping exon 45 or exon 53 for its ESSENCE study.   [...]

Following positive results obtained in Phase I/II trial of SRP-4053 (Golodirsen), Sarepta Therapeutics has announced a plan to submit a new drug application (NDA) for accelerated approval of [...]

Meeting with professor Jérôme Frenette about osteoprotegerin (OPG). We are producing a video series of interviews with Canadian researchers working on DMD. We have met many professors, and our [...]