When MDA launched the MDA National 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 see that the MDA community was eager to build connections, with interest coming from families and people of all ages, from parents of children to individuals . . .
Researchers at IONIS Pharmaceuticals are seeking individuals living with amyotrophic lateral sclerosis (ALS) with fused in sarcoma mutations (FUS-ALS) to participate in a phase 3 clinical trial to determine the efficacy of the investigational drug ION363. Drug efficacy will be determined by measuring clinical functioning and survival in trial participants. The study consists of two parts, . . .
May is Amyotrophic Lateral Sclerosis (ALS) Awareness Month, and while this past year was different — given the global pandemic we all endured — the research and support for finding new treatments never stopped. ALS is a neurodegenerative disease of the motor neurons that eventually causes muscular atrophy, paralysis, and death. The cause of ALS is understood . . .
Bella, a 15-year-old in Fort Worth, Texas, loves jellyfish, horses, anime, Billie Eilish, reading sci-fi and fantasy, and making art. But living with Friedreich’s ataxia (FA) makes it hard for her to engage in some of her favorite activities. “I can draw and paint for only a few minutes before my hand starts to hurt, so I . . .
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 . . .
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. . . .
Every year, Deborah and Jeff Corbett went to their son Josh’s annual checkup with a set of questions. They checked on Josh’s chronic ear troubles and surgeries, inquired about his lagging motor skills, and at his 4-year visit, they asked about his peculiar running gait. When he was 5, they mentioned Josh had difficulty getting . . .
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 . . .