1. On the path to a Duchenne muscular dystrophy therapy.
- Author
-
Fallon J
- Subjects
- Amyotrophic Lateral Sclerosis genetics, Amyotrophic Lateral Sclerosis physiopathology, Biomedical Research trends, Cytoskeletal Proteins metabolism, Drug Design, Dystrophin metabolism, Exons drug effects, Humans, Muscular Dystrophy, Duchenne genetics, Muscular Dystrophy, Duchenne physiopathology, Promoter Regions, Genetic, Rhode Island, Utrophin metabolism, Amyotrophic Lateral Sclerosis drug therapy, Biglycan pharmacology, Cytoskeletal Proteins drug effects, Drug Industry trends, Dystrophin drug effects, Molecular Targeted Therapy trends, Muscular Dystrophy, Duchenne drug therapy, Utrophin drug effects
- Abstract
Duchenne Muscular Dystrophy (DMD) is a devastating inherited disease of children with no effective therapies. Here I discuss the landscape for new treatments and the history, current status and prospects for our work developing recombinant biglycan as DMD therapy.
- Published
- 2013