Archives
FDA Approves Sarepta Therapeutics’ Vyondys 53 for Treatment of DMD Amenable to Exon 53 Skipping
On Dec. 12, the US Food and Drug Administration (FDA) granted accelerated approval to golodirsen (Vyondys 53) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon…
New MDA Educational Programs for Clinicians Offer a Detailed View on Selected Topics in Neuromuscular Disease
MDA’s Medical Education department was established in response to the growing need for clinician education in neuromuscular disease (NMD). We are pleased to present four webinars that cover newborn screening…
FDA Approves Aquestive Therapeutics’ Exservan for the Treatment of ALS
On Nov. 25, the US Food and Drug Administration (FDA) approved riluzole oral film (Exservan) for the treatment of amyotrophic lateral sclerosis (ALS). Exservan is an oral film formulation of…
Tags: Drug Approval, Research Advances
Kadimastem Announces Positive Interim Results from Phase 1/2a Study of AstroRx in ALS
Israel-based Kadimastem Ltd. announced positive interim results from the first cohort of its phase 1/2a clinical trial evaluating the safety, tolerability, and preliminary efficacy of AstroRx in patients with amyotrophic…
FDA Accepts Roche Genentech’s NDA for Risdiplam for the Treatment of SMA
On Nov. 25, Genentech, a member of the Roche Group, announced that the US Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review…
Zogenix Announces Positive Results from Pivotal Study of Investigational Treatment MT1621 for TK2 Deficiency
At the 24th Annual Congress of the World Muscle Society (WMS) held in October 2019 in Copenhagen, Denmark, Zogenix presented positive results from its pivotal phase 2 retrospective clinical trial (RETRO)…
Tags: Clinical Trials, Grants, Research, Research Advances
Santhera Pharmaceuticals Releases Letter to DMD Community Announcing Temporary Suspension of Enrollment in BreatheDMD Expanded Access Program
On Nov. 19, Santhera Pharmaceuticals released a letter to the Duchenne muscular dystrophy (DMD) community announcing its decision to suspend enrollment of new participants in the BreatheDMD Expanded Access Program (EAP) until…
Tags: Clinical Trials, Research
NALC to Deliver the Cure for MDA on #GivingTuesday
Since 1952, the National Association of Letter Carriers, the nationwide union of US postal delivery workers, has supported MDA as its charity of choice — and raised more than $100…
Tags: Fundraising, MDA Care Centers, Summer Camp
Fire Fighter, Father, Friend: JR Ortiz Bolsters MDA, From Boston to New York City
Santos “JR” Ortiz hadn’t always dreamed of becoming a fire fighter. He didn’t play on fire trucks as a kid, or even know much about fire departments. But when he…
Tags: Ambassadors, Summer Camp, Team Momentum
Roche Genentech Announces Positive Results from Pivotal Study of Risdiplam in SMA Types 2 and 3
On Nov. 10, Genentech, a member of the Roche Group, announced positive results from the second part of its pivotal phase 2/3 SUNFISH clinical trial assessing risdiplam in patients with…