Biogen Announces Positive Long-Term Results from Phase 2 Study of Spinraza to Treat SMA in Pre-Symptomatic Infants

Biogen announced positive long-term results from its ongoing phase 2 (NURTURE) clinical trial evaluating Spinraza (nusinersen) for treating spinal muscular atrophy (SMA) at the Cure SMA Annual Conference held June 28 through July 1 this year. New data from the NURTURE study demonstrated that after almost four years, infants who were treated with Spinraza before developing symptoms of SMA . . .

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The ALS Association, ALS Finding A Cure, and MDA Co-Fund Clinical Trial Grant to Study Restoring Immune System Function to Treat ALS

The ALS Association, ALS Finding a Cure (ALSFAC), and MDA announced they have jointly awarded a clinical trial grant totaling more than $2.5 million over two-and-a-half years to leading investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital. The principal investigator is Stanley Appel, MD, co-director of Houston Methodist Neurological Institute, chair of . . .

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Scholar Rock Announces Positive Results from Phase 1 Study of SRK-015 to Treat SMA

Scholar Rock Holding Corporation announced positive results from its phase 1 clinical trial evaluating SRK-015, a therapy for treating spinal muscular atrophy (SMA), at the Cure SMA Annual Conference held June 28 through July 1 this year. Consistent with previously announced interim findings in February, the final results showed positive safety, pharmacodynamic, and pharmacokinetic data in healthy volunteers, supporting . . .

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DMD Patients Invited to Participate in a Survey About Symptoms and Daily Life

Researchers at the University of Rochester in New York seek patients diagnosed with Duchenne muscular dystrophy (DMD) who are interested in helping to develop disease-specific patient-reported outcome measures for future clinical trials. This study will help to determine the most critical symptoms of children, young adults, and adult patients with DMD, and as a result, . . .

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Clinical Trial Alert: Alexion Pharmaceuticals Seeks Participants for a Phase 3 Pediatric Myasthenia Gravis Study

Researchers at Alexion Pharmaceuticals are looking for children with generalized myasthenia gravis (gMG) to participate in an open-label Phase 3 study. The goal of the study is to evaluate the safety and efficacy of eculizumab (Soliris) in pediatric patients. This therapy may improve muscle strength by reducing inflammation caused by the immune system. All participants . . .

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Clinical Trial Alert: Biogen Seeks Participants for a Phase 3 SOD1-ALS Study

Researchers at Biogen are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in the VALOR Phase 3 study designed to help researchers evaluate the effects of BIIB067 on disease progression in patients with ALS caused by superoxide disumtase 1 (SOD1) mutation (SOD1-ALS). In ALS, motor neurons degenerate or die and stop sending messages . . .

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Progress in Motion: MDA’s 2019 Clinical & Scientific Conference Heralds New Directions for Neuromuscular Disease Treatment

In April, MDA held its first ever combined Clinical & Scientific Conference, themed “Progress in Motion,” in Orlando Fla. Clinicians, scientists, policymakers, nonprofit, and industry leaders convened for a dynamic and informative five days in Orlando, Fla. With more than 1,200 attendees, 23 sessions, 136 presentations, and 300 posters, this was MDA’s largest conference ever. . . .

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FDA Grants Orphan Drug Designation to Acceleron Pharma’s ACE-083 Muscle Growth Drug for Charcot-Marie-Tooth Disease

On Mar. 5, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug designation to Acceleron Pharma’s ACE-083, a locally acting muscle agent, for treating Charcot-Marie-Tooth disease (CMT). Delivered by intramuscular injection, ACE-083 is based on the naturally occurring protein follistatin and is designed to enhance the body’s own promoters of muscle growth, specifically in . . .

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Clinical Trial Alert: Researchers at Neurogene Seek Participants for a CMT4J Natural History Study

Researchers at Neurogene are seeking participants living with Charcot-Marie-Tooth disease (CMT) type 4J to participate in a natural history study. This study aims to better understand disease course so researchers will be able to determine clinically meaningful outcome measures for use in future clinical trials. This five-year study will consist of 10 visits (one visit every six months). . . .

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