Clinical Trial Alert: Observational Study to Identify Biomarkers in Individuals Receiving Radicava for ALS

Researchers at Mitsubishi Tanabe Pharmaceutical America (MTPA) are seeking individuals living with amyotrophic lateral sclerosis (ALS) to participate in a six-month observational study. This study is designed to identify biomarkers to serve as quantifiable, biological, non-clinical measures of Radicava (edaravone) effects on ALS. Observational measures might include assessment of vitals; blood and urine collection; safety assessments; use . . .

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Clinical Trial Alert: Phase 3 Study of Zilucoplan in Individuals With Generalized Myasthenia Gravis

Researchers at Ra Pharmaceuticals (now part of UCB BioSciences) are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study to confirm safety, tolerability, and efficacy of the investigational drug Zilucoplan. This therapy may improve muscle function by blocking the autoimmune reaction that destroys the junction between nerves and muscles in . . .

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FDA Approves Sarepta Therapeutics’ Amondys 45 for Treatment of DMD Amenable to Skipping Exon 45

On Feb. 25, the US Food and Drug Administration (FDA) granted accelerated approval to casimersen (Amondys 45) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 45. It is the fourth exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous . . .

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Clinical Trial Alert: Phase 3 Study of Rozanolixizumab in Individuals With Generalized Myasthenia Gravis

Researchers at UCB BioSciences are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study. The objective of the study is to confirm the clinical efficacy and to assess safety and tolerability of rozanolixizumab. The therapy may help reduce the typical signs and symptoms of gMG, which are caused by autoantibodies . . .

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How Expedited Drug Approval Impacts the Neuromuscular Disease Treatment Landscape

The urgency of the COVID-19 pandemic has transformed the drug-development landscape, driving rapid development, US review, and, in one case, new therapeutic approval. To bring critical drugs to market, the US Food and Drug Administration (FDA) is utilizing a special emergency program announced in April 2020 to accelerate approval of promising coronavirus therapies, an initiative . . .

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Research Study Alert: Survey on the Mental Health Experience of Parents and Guardians of Children With DMD

Researchers at the University of Pittsburgh invite parents and guardians of children with Duchenne muscular dystrophy (DMD) to participate in a study that investigates whether they utilize mental health resources and if they have encountered any barriers to accessing such resources.  For this study, researchers at the University of Pittsburgh will ask participants to take a . . .

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Research Study Alert: LGMD2I Natural History Study

Researchers at ML Bio Solutions are seeking individuals living with limb-girdle muscular dystrophy type 2I (LGMD2I, LGMD R9-FKRP-related) to participate in a 12-month natural history study. The objective of this observational study is to identify appropriate endpoints that will be used in the phase 3 clinical trial of BBP-418 (ribitol). Data gathered may include: Fine-needle muscle biopsy . . .

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Once a Wild Idea, Successful First-Generation Exon-Skipping Therapies Pave the Way for Personalized Treatments

In 1996, MDA provided a small starter grant to Dr. Steve Wilton, a primary investigator at the University of Western Australia in Perth, for research into what scientists then thought was a radical process to address the effects of certain gene mutations. A quarter of a century later, this process — exon skipping — has . . .

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Scholar Rock Announces Positive Results from Interim Analysis of TOPAZ Phase 2 Study of SRK-015 to Treat SMA

On Oct. 27, Scholar Rock announced positive results from an interim analysis of its phase 2 clinical trial evaluating SRK-015, a therapy for treating spinal muscular atrophy (SMA). After six months of treatment with SRK-015, patients with SMA types 2 and 3 demonstrated significant motor function improvements, as measured by the Hammersmith scale, with higher dose . . .

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Research Study Alert: People With DMD Invited to Participate in a Survey About Symptoms

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

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