Barcelona, Spain – Researchers from the Neuromuscular Diseases Group and the Dementia Neurobiology Group of the Sant Pau Research Institute (IR Sant Pau) and the Memory Unit of the Sant Pau Hospital, led by neurologist Dr. Ricard Rojas-García, have identified a new mutation in the ARPP21 gene that could be...
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Santa Susana High School graduate Sean Shrager doesn’t believe in giving up. Though he was diagnosed with Duchenne muscular dystrophy (DMD) when he was 3 years old, Sean has never let his disease become an excuse for not attaining his goals. In fact, it has only motivated him to work...
Pratteln, Switzerland – Santhera Pharmaceuticals (SIX: SANN) and ReveraGen Biopharma announce new clinical data of 2.5-year treatment outcome with vamorolone in patients with Duchenne muscular dystrophy (DMD). These Phase 2a long-term treatment data demonstrate a maintenance of treatment effect, equivalent to a delay of about two years in decline for...
Pratteln, Switzerland, and Rockville, MD, USA – Santhera Pharmaceuticals (SIX: SANN) and ReveraGen BioPharma, Inc (US: private) announce positive key results from the VISION-DMD study, demonstrating robust efficacy across multiple efficacy endpoints and favorable safety and tolerability of vamorolone in the treatment of patients with DMD. VISION-DMD is a pivotal...
Santhera Announces Completion of First 6-Month Period of Pivotal VISION-DMD Trial with Vamorolone in Duchenne Muscular Dystrophy Pratteln, Switzerland Santhera Pharmaceuticals (SIX: SANN) announces that the last patient has completed the last visit for the first period of the placebo-controlled pivotal VISION-DMD study with vamorolone in patients with Duchenne muscular...
Pratteln, Switzerland – Santhera Pharmaceuticals (SIX: SANN) announces that the China National Medical Products Administration (NMPA) has accepted for priority review the new drug application (NDA) for vamorolone in Duchenne muscular dystrophy (DMD) which was submitted by Sperogenix Therapeutics, Santhera’s specialized rare disease partner for China. The Center for Drug...
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...
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...