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.
Careful assessments of motor, cognitive and language function in very young children with Duchenne muscular dystrophy (DMD) show these children lag behind their age-matched peers well before obvious symptoms of the disease appear.
Permanent repair of a faulty gene has long been a goal of researchers working to develop gene-based therapies. But many current gene modification strategies that have entered clinical trials have been based on temporary forms of gene correction — treatments that will need to be given frequently throughout a person's life.