Treatments

Sarepta Therapeutics reports sustained functional improvement two years after treatment with SRP-9001, its investigational micro-dystrophin gene therapy for Duchenne muscular dystrophy. [...]

Santhera exercises option to obtain worldwide rights of Vamorolone for Duchenne muscular dystrophy (DMD) and All Other Indications La Force is happy to share this press release provided [...]

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, [...]

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 [...]

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 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 [...]

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 [...]

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, [...]

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 [...]

PTC Therapeutics Announces First Publication of Real-World Data Showing Translarna™ (ataluren) Significantly Preserves Ability to Walk for Longer in Children with Duchenne Muscular Dystrophy [...]