Charles Gersbach, assistant professor at Duke University in Durham, N.C., was awarded an MDA research grant totaling $300,000 over a period of three years to develop a new approach for gene therapy in Duchenne muscular dystrophy (DMD).
Moon-Chang Choi, a postdoctoral researcher at Duke University in Durham, N.C., was awarded an MDA development grant totaling $120,000 over a period of two years to study one of the molecular controls regulating muscle repair.
The Muscular Dystrophy Association has awarded $120,000 to Cambridge, Mass.-based Catabasis Pharmaceuticals. The award is part of a strategic partnership under which the pharmaceutical company will test two compounds — CAT-1004 and CAT-1040 — in the mdx research mouse model of Duchenne muscular dystrophy (DMD).
It's been widely accepted that the mechanism by which the experimental drug ataluren appears to benefit walking ability in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) is that it causes "read-through" of premature stop codons — genetic instructions that cause cells to stop making a protein before the process is complete.
Eteplirsen, an experimental exon-skipping drug designed to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show sustained benefit on walking distance through week 84 of a phase 2b, 12-person study.