Pfizer doses the first participant with Its gene therapy
Pfizer doses the first participant in phase 3 study for Duchenne muscular dystrophy (DMD) investigational gene therapy. January 07, 2021, > Original press release < Pfizer Inc. announced [...]
Pfizer receives FDA fast track designation for DMD gene therapy
Pfizer receives FDA fast track designation for Duchenne muscular dystrophy (DMD) investigational gene therapy. Official press release > NEW YORK–(BUSINESS WIRE) Pfizer Inc. announced [...]
Sustained functional improvement with micro-dystrophin gene treatment
Sarepta Therapeutics reports sustained functional improvement two years after treatment with SRP-9001, its investigational micro-dystrophin gene therapy for Duchenne muscular dystrophy. [...]
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 [...]
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., [...]
SIDEROS, the largest ongoing trial in Duchenne
Santhera Completes Enrollment of Phase 3 SIDEROS Study with Puldysa® (Idebenone) in Duchenne Muscular Dystrophy (DMD) Pratteln, Switzerland, May 20, 2020 Original press release Santhera [...]
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 [...]
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 [...]
