Author: Sujatha Gurunathan

Research

Research Study Alert: Study of New Device for use in Adolescents Living with Autoimmune Neuromuscular Conditions

Sujatha Gurunathan

Researchers at Worrell are seeking adolescents (ages 12 – 17) with the following conditions: neuromyelitis optica spectrum disorder (NMOSD), myasthenia gravis (gMG), myelin oligodendrocyte glycoprotein antibody disease (MOG-AD), or autoimmune encephalitis (AE), to participate in a research study to test the safety and efficacy of a new syringe device being developed to administer a drug to . . .

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Young Adults, Research

Research Study Alert: Survey for Young Adults with Muscular Dystrophy

Sujatha Gurunathan

Researchers at the Hospital for Sick Children in Toronto, Canada are seeking young adults living with muscular dystrophy to participate in a survey about their interest in and experiences receiving genetic information. Participants will be required to complete a 10-15 minute online survey that collects largely quantitative data, as well as some open-ended responses. After completing . . .

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Research

Research Study Alert: Natural History Study in Males with BMD

Sujatha Gurunathan

Researchers at the University of Florida are seeking adult males living with Becker muscular dystrophy (BMD) to participate in a three-year natural history study. The objective of this observational study is to gain a better understanding of the course of BMD and to identify biomarkers for BMD. This study does not involve an intervention. Enrolled participants will . . .

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Research

Clinical Trial Alert: Phase 2/3 Study of Tideglusib (AMO-02) in Children and Adolescents with Congenital Myotonic Dystrophy

Sujatha Gurunathan

Researchers at AMO Pharma Ltd are seeking children and adolescents living with congenital myotonic dystrophy (DM1), also known as Steinert’s disease, to participate in a phase 2/3 clinical trial (REACH CDM) to evaluate efficacy of the investigational drug tideglusib (AMO-02) to treat congenital DM1. Tideglusib is designed to disrupt the RNA repeat that causes congenital DM1, . . .

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Research

Clinical Trial Alert: Early Phase Study of SRP-9001 in Boys with DMD

Sujatha Gurunathan

Researchers at Stanford Neuroscience Health Center are seeking 3-year-old boys living with Duchenne muscular dystrophy (DMD), particularly in California, to participate in an early phase clinical trial (a new cohort of the ENDEAVOR trial) to evaluate efficacy of Sarepta’s investigational gene replacement therapy SRP-9001 to treat DMD. SRP-9001 uses an adeno-associated virus (AAVrh74) to introduce a . . .

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News, Research

Muscular Dystrophy Association Awards 18 Grants Totaling Over $1.6 Million for Neuromuscular Disease Research

Sujatha Gurunathan

On Oct. 28, 2021 the Muscular Dystrophy Association (MDA) announced the awarding of 18 new grants totaling over $1.6 million toward neuromuscular disease (NMD) research. These new grants represent a continued commitment by MDA to fund groundbreaking research that will one day lead to treatments and cures for the diseases in its program. The newly . . .

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Adults, News, Research

Research Study Alert: Observational Study of Clinical and Electrophysiological Outcomes in Adults with CMT

Sujatha Gurunathan

Researchers at Ohio State University are seeking adults living with Charcot-Marie-Tooth disease type 1 (CMT-1) and type 2 (CMT-2) to participate in a one-month observational study (CMT Establish). This study is designed to assess whether individuals with CMT-1 and CMT-2 exhibit altered communication between nerves and muscles, known as neuromuscular junction (NMJ) transmission, compared to healthy . . .

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News, Research, Uncategorized

Clinical Trial Alert: Phase 3b Study of Oral Edaravone in Adults with ALS

Sujatha Gurunathan

Researchers at Mitsubishi Tanabe Pharma Development America, Inc. (MTPA) are seeking adults living with amyotrophic lateral sclerosis (ALS) to participate in a phase 3b clinical trial to evaluate the safety and efficacy of oral edaravone to treat ALS. Edaravone (brand name Radicava) was designed to help protect cells from damage caused by free radicals, and was . . .

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News, Research

Capricor Announces Positive Results from its Phase 2 Study of CAP-1002 to Treat DMD

Sujatha Gurunathan

On September 24, Capricor Therapeutics announced positive results at the World Muscle Society Virtual Congress from its phase 2 HOPE-2 trial of the investigational therapy CAP-1002 for treatment of people in advanced stages of Duchenne muscular dystrophy (DMD). The primary and secondary endpoints of the study, the improvement of upper limb and cardiac function, were met, . . .

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News

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

Sujatha Gurunathan

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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