Martin Childers, PhD, DO, chief medical officer at Asklepios BioPharmaceutical Inc. in North Carolina, was awarded an MDA research grant totaling $192,500 over one year to perform pre-clinical studies using an adeno-associated virus (AAV) to deliver a gene therapy for limb-girdle muscular dystrophy type 2I (LGMD2I). Limb-girdle muscular dystrophies are a diverse group of disorders . . .
The National Limb Girdle Muscular Dystrophy Conference, scheduled Aug. 30 to Sept. 2 at the Hyatt Regency in Chicago, will highlight all forms of limb-girdle muscular dystrophy (LGMD) in a gathering of researchers, neurologists, biotechs, and patients. A project and program of The Speak Foundation, a nonprofit focused on improving the quality of life for . . .
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 . . .
The Muscular Dystrophy Association (MDA) is pleased to announce a collaboration with the Broad Institute of MIT and Harvard, which aims to bring genetic diagnoses to more individuals living with limb-girdle muscle weakness. The goal of the LGMD Rare Genomes Project is to bring the most advanced genetic techniques, including whole-genome sequencing and genetic variation . . .
On Feb. 27, Sarepta Therapeutics announced positive interim results of a phase 1/2a clinical trial for MYO-101, a gene therapy candidate developed by Myonexus Therapeutics for patients living with limb-girdle muscular dystrophy type 2E (LGMD2E), also known as beta-sarcoglycanopathy. The first three patients dosed demonstrated significant expression of the protein beta-sarcoglycan in the muscle fiber, . . .
On Dec. 23, 2018, the MDA office of Minnesota and the Dakotas received its biggest gift ever — and it came as a surprise from one man who felt connected to MDA’s mission. Steven Williams, a native of Clarkfield, Minn. and a Vietnam War veteran, graduated from college in the mid-1970s with a degree in . . .
On Jan. 2, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug Designation for MYO-102, an investigational gene therapy for limb-girdle muscular dystrophy type 2D (LGMD2D), also known as alpha-sarcoglycanopathy. Myonexus Therapeutics has licensed the technology from Nationwide Children’s Hospital, which holds the Investigational New Drug (IND) application for MYO-102. Treatment using gene therapy, . . .
Sept. 30th is Limb-Girdle Muscular Dystrophy Awareness Day, which celebrates individuals living with the more than 30 different subtypes of Limb-girdle muscular dystrophy (LGMD). MDA has a long-standing commitment to LGMD — we’ve invested more than $59 million in LGMD-specific research since 1950, including more than $2.8 million in current active grants. MDA Scientific Portfolio . . .
Every year on Sept. 30, people around the world wear lime green and come together online and in their communities to celebrate Limb-Girdle Muscular Dystrophy (LGMD) Awareness Day. Now four years old, LGMD Awareness Day has been embraced by the LGMD patient community as a way to raise awareness and honor individuals living with the . . .
Originally published in the Hartford Courant on July 22, 2018. To see the original article, click here. For many years I was consumed by worry and bitterness, thinking I was the only one who was given an unfair lot in life. My body, once athletic and fit, was weakening from the uncompromising progression of an adult-onset neuromuscular disease . . .