Pratteln, Switzerland – Santhera Pharmaceuticals (SIX: SANN) announces positive topline results from its long-term Phase 4 LEROS study with Raxone® (idebenone) in the treatment of Leber’s hereditary optic neuropathy (LHON). The primary endpoint, proportion of eyes with clinically relevant benefit after 12 months treatment with Raxone versus untreated patients from an...
treatment News
Pratteln, Switzerland – Santhera Pharmaceuticals (SIX: SANN) announces positive results from its multiple ascending dose Phase 1b study with lonodelestat, a potent inhibitor of human neutrophil elastase (hNE), in patients with cystic fibrosis (CF). Lonodelestat is a potent and selective peptide inhibitor of human neutrophil elastase (hNE), currently being developed...
Pratteln, Switzerland – Santhera Pharmaceuticals (SIX: SANN) announces the publication of the paper “Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy” in the peer-reviewed journal Neurology. The publication reports the results of the 48-week treatment with vamorolone in patients with DMD in the VISION-DMD...
Pratteln, Switzerland — Santhera Pharmaceuticals (SIX: SANN) announces that has submitted a marketing authorization application (MAA) to the UK Medicines and Healthcare products Regulatory Agency (MHRA) for vamorolone for the treatment of Duchenne muscular dystrophy (DMD). In parallel to the MAA submission to the UK MHRA, Santhera is currently preparing...
Pratteln, Switzerland – The European Commission (EC) granted marketing authorization to vamorolone for the treatment of patients with Duchenne muscular dystrophy (DMD), who are 4 years of age and above, according to a press release from Santhera, the developers of the treatment. Vamorolone will be sold under the brand name...
Roswell, Ga, Dublin and Hamilton, Bermuda — Saol Therapeutics (“Saol”), a privately held, clinical-stage pharmaceutical company, announced the signing of an agreement with Medosome Biotec to provide Saol the rights to use their patented genotype test for all potential indications where SL-1009 might be used as a therapy. The test...
CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive data from Part B of the MOMENTUM study (Study SRP-5051-201), a global, Phase 2, multi-ascending dose clinical trial of SRP-5051 (vesleteplirsen) that enrolled patients aged 8 to 21 years. SRP-5051 is a...
CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the U.S. Food and Drug Administration (FDA) has accepted and filed the Company’s efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) (the “Efficacy Supplement”). The goals of the...
CAMBRIDGE, Mass. – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from Part A of the MOMENTUM study (Study 5051-201), a global, Phase 2, multi-ascending dose clinical trial of SRP-5051, its next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with...
CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, today announced submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) to expand its labeled indication as follows “[ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD)...