Archives
Clinical Trial Alert: Phase 3 Study of Pamrevlumab (FG-3019) in Combination with Systemic Corticosteroids in Ambulatory Boys with DMD
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…
Clinical Trial Alert: Phase 2 Study of Pitolisant for Treatment of Excessive Daytime Sleepiness in Adults with Myotonic Dystrophy, Type 1 (DM1)
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…
Clinical Trial Alert: Phase 1b and Open-Label Extension Study of ASP0367 in Boys with DMD
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…
Spotting Neuromuscular Disease in Adults
After years of denial, it was the stairs to his second-floor college apartment that forced Chris Anselmo to finally face his limb-girdle muscular dystrophy (LGMD) in 2009. “I remember moving in, trying…
Tags: Healthcare, Research, Resources
The Benefits of Getting Kids Involved in Adaptive Sports
When Kristine Welker’s son was young, he wanted to be as involved in sports as everyone else in the family — and that meant a high level of activity, from…
Inclusion in STEM: A Discussion with General Motors
According to the National Science Foundation, individuals living with a disability, such as neuromuscular disease, are underrepresented in the fields of science, technology, engineering, and math (STEM). Yet STEM-based careers are growing…
Tags: Ambassadors, College, Education, Employment, Parenting
MDA Care Specialist Support for MDA Care Center Visits
MDA Care Specialists are dedicated to assisting families who visit MDA Care Centers by providing the right resources, answering questions, and helping navigate processes. After your visit to an MDA…
Biogen’s Early Access Program for Tofersen is Now Open for Individuals with Rapidly Progressing SOD1-ALS
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…
Tags: Clinical Trials, Drug Approval, Drug Development, Healthcare, Innovation, Research, Research Advances
Clinical Trial Alert: Phase 3 Study of Rozanolixizumab in Adults with Generalized Myasthenia Gravis
Researchers at The Neurological Institute, PA, located in Charlotte, North Carolina, are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 3 clinical trial to evaluate efficacy…
MDA Peer Connections Helps MDA Families Find Others with Shared Experiences
When MDA launched the MDA Peer Connections program last year, there was no doubt it would be invaluable for fostering relationships, especially during the pandemic. It didn’t take long to…