In October, the U.S. Food and Drug Administration (FDA) approved Agamree (vamorolone) for treating Duchenne muscular dystrophy (DMD) patients ages 2 and older, offering them a more tolerable, but similarly efficacious, oral corticosteroid option. Clinical trial evidence indicates Agamree might ease muscle damage and minimize the side effects…

The first patient has been dosed in a pilot study evaluating vamorolone as a potential treatment for Becker muscular dystrophy (BMD). The steroid medication has been under development by ReveraGen BioPharma as a possible therapy for other types of muscular dystrophy. Now, a Phase 2 trial…

Santhera Pharmaceuticals and ReveraGen BioPharma will seek approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD) in the U.S. this year, with a filing expected to be completed by the end of June. The submission of the companies’ rolling new drug application (NDA) to the U.S. Food and Drug Administration…

Santhera Pharmaceuticals has entered into an agreement with SEAL Therapeutics, a spin-off of the Biozentrum of the University of Basel, that is expected to further develop a potential gene therapy for LAMA2-deficient congenital muscular dystrophy (LAMA2 MD). The new company’s gene therapy technology builds on previous research…

Treatment with vamorolone continued to show a good safety profile and sustained efficacy across functional assessments after almost one year in boys with Duchenne muscular dystrophy (DMD), according to 48-week results of the Phase 2b VISION-DMD clinical trial. Data also showed no loss of efficacy in boys who switched…

Santhera Pharmaceuticals and ReveraGen BioPharma are gearing up to submit a new drug application (NDA) asking for U.S. approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD). The companies announced Tuesday they had completed a pre-NDA meeting with the U.S. Food and Drug Administration (FDA).

Top-line data from the VISION-DMD Phase 2b clinical trial evaluating vamorolone in boys with Duchenne muscular dystrophy (DMD), showed efficacy across primary and secondary study goals. The investigational therapy was also well-tolerated with a…

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Long-term treatment with the experimental steroid therapy vamorolone safely and effectively delays motor function decline in boys with…

The final patient has finished the first, six-month portion of the pivotal VISION-DMD Phase 2b clinical trial, assessing the anti-inflammatory steroid therapy vamorolone in boys with Duchenne muscular dystrophy (DMD). Top-line data from this part of…

Santhera Pharmaceuticals has decided to stop SIDEROS, its Phase 3 trial assessing the effectiveness of Puldysa (idebenone) at delaying respiratory decline in patients with Duchenne muscular dystrophy (DMD) on a stable glucocorticoid dose. An interim data analysis of the study (NCT02814019), which was fully enrolled, determined that…