Beginning in the early 1950s, when public awareness and understanding of muscular dystrophy and related neuromuscular diseases were extremely limited, MDA began calling upon individuals living with these diseases to serve as National Ambassadors, telling their personal stories and inspiring support of MDA. More than 40 MDA National Ambassadors, including children and adults, have met U.S. . . .
Researchers at Apellis Pharmaceuticals are seeking individuals living with sporadic amyotrophic lateral sclerosis (ALS) to participate in a two-year phase 2 trial (MERIDIAN) to determine the efficacy of the investigational drug pegcetacoplan. Pegcetacoplan is designed to reduce the activity of the complement system (a component of the immune system), and potentially slow the progression of ALS. . . .
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 Audentes Therapeutics, an Astellas company, are seeking individuals living with late-onset Pompe disease (LOPD) to participate in a phase 1/2 study to confirm safety and efficacy of the investigational drug AT845. This gene-replacement therapy may offer the benefit of long-term improvement of motor and respiratory function and quality of life in adults living with . . .
Researchers at Mitsubishi Tanabe Pharmaceutical America (MTPA) are seeking individuals living with amyotrophic lateral sclerosis (ALS) to participate in a six-month observational study. This study is designed to identify biomarkers to serve as quantifiable, biological, non-clinical measures of Radicava (edaravone) effects on ALS. Observational measures might include assessment of vitals; blood and urine collection; safety assessments; use . . .
Researchers at Ra Pharmaceuticals (now part of UCB BioSciences) are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study to confirm safety, tolerability, and efficacy of the investigational drug Zilucoplan. This therapy may improve muscle function by blocking the autoimmune reaction that destroys the junction between nerves and muscles in . . .
Researchers at UCB BioSciences are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study. The objective of the study is to confirm the clinical efficacy and to assess safety and tolerability of rozanolixizumab. The therapy may help reduce the typical signs and symptoms of gMG, which are caused by autoantibodies . . .
Researchers at the University of Pittsburgh invite parents and guardians of children with Duchenne muscular dystrophy (DMD) to participate in a study that investigates whether they utilize mental health resources and if they have encountered any barriers to accessing such resources. For this study, researchers at the University of Pittsburgh will ask participants to take a . . .
Researchers at ML Bio Solutions are seeking individuals living with limb-girdle muscular dystrophy type 2I (LGMD2I, LGMD R9-FKRP-related) to participate in a 12-month natural history study. The objective of this observational study is to identify appropriate endpoints that will be used in the phase 3 clinical trial of BBP-418 (ribitol). Data gathered may include: Fine-needle muscle biopsy . . .
Living with neuromuscular disease (NMD) involves specific challenges — some physical, some strategic, some structural — that can create stress and contribute to changes in both physical and mental health. Can meditation, mindfulness, and other relaxation practices help? Yes, says Paige Lembeck, PhD, a pediatric psychologist and assistant professor of Clinical Child Psychology at the . . .