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 . . .
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 . . .
On Aug. 6, the US Food and Drug Administration (FDA) granted accelerated marketing approval to avalglucosidase alfa (Nexviazyme) for the treatment of people 1 year of age and older living with late-onset Pompe disease. It is the second approved drug to treat Pompe disease. Nexviazyme will be made available in the US and marketed by Sanofi . . .
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. . . .
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 . . .
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 . . .
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 . . .
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 . . .
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 . . .
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 . . .
