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 . . .
The Muscular Dystrophy Association recently awarded an MDA clinical research network grant to Nicholas Johnson, vice chair of research and associate professor of neurology at Virginia Commonwealth University in Richmond, to establish the Limb-Girdle Muscular Dystrophy (LGMD) Clinical Research Network. The investment, totaling $700,000 over two years, supports seven centers with expertise in LGMD research . . .
Whenever Paul Robertson and his Fishing for Muscular Dystrophy (FFMD) team pull into a truck stop, they know it might be a while before they are able to leave. Although there to pump fuel, the caravan of trucks and boats adorned with the FFMD and MDA logos are such a big hit with fellow motorists . . .