FDA Acceptance of Casimersen
Sarepta Therapeutics Announces FDA Acceptance of Casimersen (SRP-4045) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45 Original press release, [...]
Viltolarsen, exon 53 skipping therapy, approved
N.S. Pharma’s VILTEPSO™ (viltolarsen) injection now FDA-Approved in the U.S. for the treatment of Duchenne muscular dystrophy in patients amenable to exon 53 skipping therapy. Patients [...]
Translarna: Update for Non-Ambulatory Patients with DMD
PTC Therapeutics Announces that The Committee for Medicinal Products for Human Use (CHMP) Recommendation of Translarna ™ (ataluren) Label Update for Non-Ambulatory Patients with Duchenne Muscular [...]
Sarepta is seeking the approval of casimersen for DMD patients
Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045) for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45 Casimersen [...]
Positive Safety and Efficacy Data from Micro-Dystrophin Gene Therapy
Sarepta Therapeutics Announces Positive Safety and Efficacy Data from the SRP-9001 Micro-Dystrophin Gene Therapy Trial Published in JAMA Neurology Press release here > CAMBRIDGE, Mass., [...]
Press release from Pfizer about gene therapy
Pfizer’s new phase 1b results of gene therapy in ambulatory boys with Duchenne muscular dystrophy (DMD) support advancement into pivotal phase 3 study Read the press release here. Friday, [...]
Latest edition of the Catabasis Connection newsletter
La Force is happy to share the latest edition of the Catabasis Connection newsletter with updates on edasalonexent and information that Catabasis shared at the MDA Virtual Poster Session. Earlier [...]
COVID-19 for people affected by Duchenne or Becker #3
What we know about COVID-19 part 3 What do we know about COVID-19 for people affected by Duchenne or Becker? Due to the increasing concerns regarding the COVID-19 virus for people with Duchenne [...]
COVID-19 for people affected by Duchenne or Becker #2
What we know about COVID-19 part 2 What do we know about COVID-19 for people affected by Duchenne or Becker? Due to the increasing concerns regarding the COVID-19 virus for people with Duchenne [...]
Edasalonexent and COVID-19
La Force is happy to share the latest edition of the Catabasis Connection newsletter. As we are all faced with an unimaginable situation, Catabasis wanted to reach out and share information in [...]
