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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What’s #GivingTuesday All About?

#GivingTuesday, annually held immediately after the Black Friday and Cyber Monday holiday shopping days following Thanksgiving, falls late this year on Dec. 3. But here at MDA, we’re already planning on how we can all join together during this Season of Will to celebrate the loved ones we know impacted by muscular dystrophy, ALS, and . . .

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Roche Genentech Releases Letter to DMD Community Announcing Discontinuation of Clinical Trials Program for DMD

Today, Roche Genentech released a letter to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program. The full letter follows. Letter to the DMD community from Roche Genentech: Dear members of the Duchenne community, We are writing to share with you unfortunate news about our clinical development program . . .

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New MDA Educational Program Offers a Detailed View of SMA Treatment Landscape

MDA’s new Medical Education department was established in response to the growing need for clinician education in neuromuscular diseases. We are pleased to present our first CME-accredited webinar highlighting the treatment landscape for spinal muscular atrophy (SMA). SMA is a neuromuscular disorder that often presents as hypotonia and weakness shortly after birth. SMA is characterized . . .

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AveXis Releases Letter to SMA Community: FDA Places Partial Hold on Clinical Trials for Intrathecally Delivered AVXS-101

On Oct. 30, AveXis released a letter to the spinal muscular atrophy (SMA) community regarding a decision by the US Food and Drug Administration (FDA) to place a partial hold on clinical trials for intrathecal (IT) administration of AVXS-101. The full letter follows. The order stops enrollment of patients in the high-dose group of AveXis’ . . .

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MDA Engage Social Gathering Spotlight

Throughout the fall and winter months, MDA Engage Social Gatherings will be taking place throughout the country. These gatherings facilitate community through establishing opportunities for families, clinicians, researchers, volunteers, sponsors, supporters, and MDA staff to connect in a casual social setting. Participants tell their stories and are united within the neuromuscular community, supporting each other . . .

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Clinical Trial Alert: Amicus Therapeutics Seeks Teenage Participants for a Phase 3 Late-Onset Pompe Disease Study

Researchers at Amicus Therapeutics are looking for adolescents (age 12-18) with late-onset Pompe disease (LOPD) to participate in a phase 3 study. The goal of the study is to evaluate the safety and efficacy of ATB200/AT2221, which researchers hope may improve muscle function and respiratory function compared to the standard of care. Participants will be . . .

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Casey’s General Stores Round Up for MDA

This September, at Casey’s General Stores in 16 states across the Midwest, employees have been doing more than serving guests. They’ve been serving the MDA community, too, inviting store guests to round purchase totals up to the nearest whole dollar to support individuals and families impacted by neuromuscular disease. Now in its 13th year of . . .

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New MDA Educational Program Offers a 360-Degree View of ALS Care

MDA’s new Medical Education department was established in response to the growing need for clinician education in neuromuscular diseases. We are pleased to present our first program highlighting amyotrophic lateral sclerosis (ALS): a PowerPoint slide deck. ALS is a motor neuron disease that leads to the widespread loss of muscle use. Patients diagnosed with ALS . . .

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