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 . . .
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 . . .
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, . . .
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. . . .
Researchers at the Icahn School of Medicine at Mount Sinai are seeking disability advocates to participate in a survey about the current state of genetic counseling for the disability community. The goal of this survey is to help understand gaps in knowledge among genetic counselors regarding life with disability and to evaluate educational opportunities, such as . . .
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 . . .
Researchers at University of Rochester are seeking individuals living with Duchenne muscular dystrophy (DMD) to participate in a survey to help with development and validation of a disease-specific, patient-reported outcome measure for clinical trials involving people with DMD. Participants will need to complete an online survey and a follow-up phone interview. The survey will take approximately 20 . . .
Researchers at Johns Hopkins University School of Medicine are seeking individuals living with amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), or a related motor neuron disease to participate in a study/survey to determine if disease progression can be tracked by a smartphone application. The study uses REDCap, a secure website for medical research, to collect information . . .
Researchers at the State University of New York at Binghamton’s School of Pharmacy and Pharmaceutical Sciences are seeking participants living with Becker muscular dystrophy (BMD) to participate in a biomarker and patient-reported outcomes study. The purpose of the Becker Muscular Dystrophy Biomarker and Patient-Reported Outcomes Study is to learn more about disease progression in patients . . .