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 […]
As an early funder of Capricor Therapeutics, CureDuchenne is pleased to share that the FDA has set the PDUFA target action date for August 22, 2026 as the deadline to […]
NS Pharma, Inc. presented 4.5-year safety and efficacy data based on the open-label extension study of brogidirsen (NS-089/NCNP-02) an antisense oligonucleotide for DMD patients with mutations amenable to skipping exon […]
