Clinical Trial Alert: Phase 2 Study of Ifetroban in Individuals with DMD

Researchers at Cumberland Pharmaceuticals Inc. are seeking boys and men living with Duchenne muscular dystrophy (DMD) to participate in a phase 2 clinical trial to evaluate the safety, efficacy, and duration of effect of oral ifetroban to treat heart disease associated with DMD. Ifetroban is designed to reduce fibrosis and fat deposition in the heart and . . .

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Clinical Trial Alert: Phase 2/3 and Open-Label Extension Study of ASP0367 in Individuals with Primary Mitochondrial Myopathy

Researchers at Astellas Pharma Inc. are seeking individuals living with primary mitochondrial myopathy (PMM) to participate in a phase 2/3 clinical trial and open-label extension to evaluate safety, tolerability and preliminary efficacy of the investigational drug ASP0367 (MA-0211) to treat PMM. ASP0367 is being investigated regarding whether it can increase the number and function of the . . .

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Clinical Trial Alert: Phase 1b/2a Study of Descartes-08 in Adults with Generalized Myasthenia Gravis

Researchers at Cartesian Therapeutics are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 1b/2a clinical trial to evaluate safety, tolerability and manufacturing feasibility of the investigational drug Descartes-08 to treat gMG. Descartes-08 is designed to eliminate the aberrant plasma cells that cause gMG, potentially reducing the clinical symptoms of the disease. . . .

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Research Study Alert: Survey for Caregivers of Children with SMA to Study Treatment Decision Making

Researchers at Michigan Medicine at the University of Michigan are seeking caregivers of children living with spinal muscular atrophy (SMA) to participate in a survey about treatment decision making. Over the last five years, three treatments have been approved and are changing life expectancy and physical functioning for children with SMA. The goal of this survey . . .

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FDA approves Octapharma’s Octagam® 10% for Treatment of Adults with Dermatomyositis

On July 20, the US Food and Drug Administration (FDA) granted approval to Octagam® 10% for the treatment of adult dermatomyositis, a rare immune-mediated inflammatory disease. Octagam® 10% is the first and only intravenous immunoglobulin (IVIg) approved for dermatomyositis. Dermatomyositis is an autoimmune condition that involves inflammation of muscles and associated tissues, such as the blood vessels that supply the muscles. The . . .

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Clinical Trial Alert: Phase 3 Study of Pamrevlumab (FG-3019) in Combination with Systemic Corticosteroids in Ambulatory Boys with DMD

Researchers at FibroGen are seeking boys living with Duchenne muscular dystrophy (DMD) to participate in a phase 3 clinical trial to evaluate efficacy of the investigational drug pamrevlumab (FG-3019) in combination with systemic corticosteroids to treat DMD. Pamrevlumab is designed to decrease fibrosis in the muscles, thereby protecting the heart and lungs. This has the potential . . .

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Clinical Trial Alert: Phase 2 Study of Pitolisant for Treatment of Excessive Daytime Sleepiness in Adults with Myotonic Dystrophy, Type 1 (DM1)

Researchers at Harmony Biosciences are seeking adults living with myotonic dystrophy, type 1 (DM1) to participate in a phase 2 clinical trial and open-label extension to evaluate safety and efficacy of the investigational drug pitolisant (brand name Wakix) to treat excessive daytime sleepiness and other non-muscular symptoms of DM1. Pitolisant treatment increases the amount the chemical . . .

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Clinical Trial Alert: Phase 1b and Open-Label Extension Study of ASP0367 in Boys with DMD

Researchers at Astellas Pharma Inc. are seeking boys living with Duchenne muscular dystrophy (DMD) to participate in a phase 1b clinical trial and open-label extension to evaluate safety, tolerability and preliminary efficacy of the investigational drug ASP0367 (MA-0211) to treat DMD. ASP0367 is designed to increase the number and function of the mitochondria in cells, thereby . . .

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Biogen’s Early Access Program for Tofersen is Now Open for Individuals with Rapidly Progressing SOD1-ALS

Beginning in mid-July 2021, Biogen is opening part 1 of its early access program for the investigational therapy tofersen to individuals with amyotrophic lateral sclerosis (ALS) caused by mutation of the superoxide dismutase 1 gene (SOD1) who are specifically experiencing rapid disease progression. Tofersen is being investigated in a phase 3 clinical study for safety and efficacy, and the . . .

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