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 . . .
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 . . .
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’ . . .
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 . . .
Researchers at Amicus Therapeutics are looking for participants (age 0-18) 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 receive the . . .
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 . . .
Researchers at Momenta Pharmaceuticals are looking for adults with generalized myasthenia gravis (gMG) to participate in a phase 2 study. The goal of the study is to evaluate the safety and efficacy of nipocalimab in patients who did not respond to standard of care. This therapy may improve function by lowering the levels of antibodies . . .
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 . . .
At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with Duchenne muscular dystrophy (DMD), as well as their caregivers and loved ones, to attend the MDA Engage DMD Symposium taking place Nov. . . .
At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with Charcot-Marie-Tooth disease (CMT), as well as caregivers and loved ones, to attend the MDA Engage CMT Symposium taking place Nov. 9 in . . .
