During a late-morning session on the final day of the 2019 Clinical & Scientific Conference, five healthcare professionals — Laurie Sterling, a speech therapist at the Texas Children’s Hospital; Dr. Roxanna Bendixen, an occupational therapist and assistant professor at the University of Pittsburgh; Jennifer Chapin, a speech therapist at the University of Florida; Dr. Melissa . . .
Myasthenia gravis (MG) is an autoimmune disease, which is a disease that occurs when the immune system goes awry and produces antibodies that attack the body’s own tissues. Normally, antibodies are created to rid the body of pathogens such as bacteria, viruses, and foreign substances. In MG, antibodies attack the connection between nerve and muscle . . .
Adam Bittel, PT, DPT, PhD, a postdoctoral fellow at Children’s National Medical Center in Washington, DC, was awarded the 2019 SSSI-MDA Fellowship Award. The award, co-sponsored by Strength, Science & Stories of Inspiration (SSSI) and MDA, will provide a total of $40,000 over two years to support Dr. Bittel’s work investigating the cellular mechanisms underlying the effects . . .
Like many pre-medical students, Justin Cohen discovered along the way that what he really liked was research. However, unlike others who exchange the stethoscope for a microscope, Justin had a strikingly different motivating factor — he has been living with the disease he studies, facioscapulohumeral muscular dystrophy (FSHD), for almost as long as he can . . .
Today, MDA and Locana, a leading RNA-targeting gene therapy company, announced the award of an MDA Venture Philanthropy (MVP) grant totaling $550,000 to advance Locana’s development program for myotonic dystrophy (DM), the most common form of adult-onset muscular dystrophy. MVP is the MDA’s drug development program that is exclusively focused on funding the discovery and . . .
Researchers at the University of Rochester in New York seek patients diagnosed with Duchenne muscular dystrophy (DMD) who are interested in helping to develop disease-specific patient-reported outcome measures for future clinical trials. This study will help to determine the most critical symptoms of children, young adults, and adult patients with DMD, and as a result, . . .
Dr. Merit Cudkowicz, chief of Neurology at Massachusetts General Hospital, where the ALS Multidisciplinary Clinic in the Healey Center for ALS is associated with MDA, is dedicated to performing academic-led clinical trials in amyotrophic lateral sclerosis (ALS). Her internationally renowned research has led to advanced treatments for people with living with ALS. What role has . . .
Today, the U.S. Food and Drug Administration (FDA) approved Zolgensma (onasemnogene abeparvovac-xioi), the first gene therapy for a neuromuscular disease. Zolgensma is a one-time intravenous (into the vein) infusion for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene, . . .
Dr. Jonathan Glass is the director of the Emory ALS Center, co-chair of the Northeast ALS Consortium, and professor of Neurology and Pathology at Emory University in Atlanta. Dr. Glass’ laboratory focuses on the study of amyotrophic lateral sclerosis (ALS), investigating causes and potential therapies and cures. MDA has been a partner in research, providing . . .
It’s a hard fact that only three medications have been approved to treat amyotrophic lateral sclerosis (ALS) in nearly 25 years. Fortunately, research in ALS has exploded in the past decade, and new technologies have enabled the development of gene-targeting therapies such as gene replacement, gene silencing, and gene editing. MDA has always been committed . . .
