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 53. It is the second exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous . . .
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 (NBS) and early intervention, genetic testing, and best practices in multidisciplinary care. Our Medical Education department, together with key opinion leaders in the NMD field, . . .
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 riluzole (Rilutek), which has been available in tablet form to treat ALS for more than two decades. Exservan will be made available in the United . . .
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 lateral sclerosis (ALS). Results presented at the 7th International Stem Cell Meeting of the Israel Stem Cell Society held recently in Tel Aviv, Israel, showed that patients . . .
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 for risdiplam, an investigational therapy for the treatment of spinal muscular atrophy (SMA). Priority Review status requires the FDA to review the application and decide on whether . . .
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) testing the safety and efficacy of pyrimidine nucleoside substrate enhancement therapy (SET) MT1621 for treating thymidine kinase 2 (TK2) deficiency. The experimental therapy was developed . . .
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 the SIDEROS clinical trial is fully enrolled. The full letter follows. SIDEROS clinical trial and BreatheDMD program update: Dear Duchenne community, In February 2018, Santhera . . .
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 million through local fundraisers that have showcased workers’ creativity and dedication to MDA’s mission of transforming the lives of people living with neuromuscular disease through . . .
Genetic testing has a critical role in patients’ care as genetic information helps physicians to distinguish between types of neuromuscular disorders, informs prognosis and treatment decisions, and determines patients’ eligibility for new and emerging therapies. Selecting the right genetic test and incorporating genetic information to reach a diagnosis and formulate a treatment plan require deep . . .
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 was in his late 20s, a friend in the Boston Fire Department started talking about the job, and JR took notice. When that friend dropped . . .