The results of the Christopher Project have been published in a comprehensive, 80-page report. The creation of “The Christopher Project Report to the Myotonic Dystrophy Community” was supported by several organizations, including the Muscular Dystrophy Association (MDA), and is available for download on MDA’s website. (Printed copies are also available upon request.) What is the . . .
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 . . .
On June 7, the U.S. Food and Drug Administration (FDA) approved Emflaza (deflazacort) to expand its labeling to include patients with Duchenne muscular dystrophy (DMD) who are between 2 and 5 years old. Emflaza was approved by the FDA in February 2017 for the treatment of DMD in patients 5 years and older, making it the first drug approved . . .
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, . . .
While most visits to the doctor lead to a quick diagnosis, the same is not so for amyotrophic lateral sclerosis (ALS). On average, getting an ALS diagnosis takes 12 months — a long time, considering that on average, ALS patients live for only two to five years after diagnosis. Some might call the process a . . .
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 . . .
At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with a neuromuscular disease, as well as caregivers and loved ones, to attend MDA Engage symposia taking place across the country. MDA Engage . . .