Research Study Alert: Survey for Young Adults with Muscular Dystrophy

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 . . .

Read More

Research Study Alert: Natural History Study in Males with BMD

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 . . .

Read More

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

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, . . .

Read More

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

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 . . .

Read More

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

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 . . .

Read More

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

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 . . .

Read More

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

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, . . .

Read More

Research Study Alert: Survey for Caregivers of Individuals with SMA about Communication Abilities

Researchers at Russell Sage College are seeking parents or legal guardians of individuals with spinal muscular atrophy (SMA) to participate in a survey about the communication abilities of the affected individuals. Analysis of the current communicative behaviors used by individuals with SMA will help researchers develop communication systems that better address the needs of this population. . . .

Read More