Strongly


An expression of the live unlimited spirit, stories and voices of the MDA community

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 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 . . .

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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 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 . . .

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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) 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 . . .

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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 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 . . .

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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 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 . . .

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New MDA Educational Program for Clinicians Offers Detailed View of Genetic Testing for Neuromuscular Disorders

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 . . .

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Solid Biosciences Releases Letter to DMD Community Announcing Hold on IGNITE DMD Trial Due to a Serious Adverse Event

Today, Solid Biosciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding the decision by the US Food and Drug Administration (FDA) to place the company’s IGNITE DMD clinical trial on hold because the latest child dosed experienced a serious adverse event (SAE). The full letter follows. Letter to the Duchenne Community: Update . . .

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