Solid Biosciences has announced the first participant has been dosed in the Phase 3 IMPACT DUCHENNE trial evaluating SGT-003. The global study is now underway, with active sites in Australia […]
Entrada Therapeutics, who received early funding from CureDuchenne to advance their Endosomal Escape Vehicle Platform, today announced positive initial data from their Phase 1/2 ELEVATE-44-201 study in DMD patients amenable […]
PTC Therapeutics provided an update to the Duchenne community with additional details following the withdrawal of the ataluren New Drug Application (NDA). The update notes that a limited remaining supply […]
What Sarepta Therapeutics’ sNDA submission means for Duchenne Sarepta Therapeutics is preparing to submit supplemental new drug applications (sNDAs) to the FDA for converting the accelerated approvals of AMONDYS 45 […]
Sarepta therapeutics is seeking to enroll 25 non-ambulant individuals with Duchenne in a clinical trial evaluating the use of sirolimus as part of an enhanced immunosuppressive regimen prior to and […]
Solid Biospace Gene Therapy SGT-003, an investigational microdystrophin gene therapy for Duchenne, continues to demonstrate an encouraging safety profile and has been generally well tolerated in 40 participants treated to […]
ITF shared a community letter and presented new data, including long-term safety observations based on data from the company’s ongoing open-label extension study in patients with Duchenne muscular dystrophy treated with […]
REGENXBIO GENE THERAPY RGX-202, an investigational gene therapy for Duchenne, continues to show a favorable safety profile with no serious adverse events, no liver injury signals, and reductions in key […]
Edgewise Therapeutics, a CureDuchenne funded company, today announced positive long-term Sevasemten data that demonstrated sustained functional stabilization in Becker Muscular Dystrophy (BMD) patients through 3.5 years of treatment. This result […]
