Article
Interventions for muscular dystrophy: molecular medicines entering the clinic.
Lancet (London, England) - 28 Nov 2009
Bushby Kate, Lochmüller Hanns, Lynn Stephen, Straub Volker
Abstract excerpt
Muscular dystrophies are individually rare genetic disorders that cause much chronic disability, affecting young children and adults. In the past 20 years, more than 30 genetic types of muscular dystrophy have been defined. During this time, precise diagnosis, genetic counselling, and medical management have improved. These advances in medical practice have occurred while definitive therapies based on an improved...
Topics
- Animals
- Disease Models, Animal
- Dystrophin
- Genetic Therapy
- Humans
- Mice
- Muscular Dystrophies
- Mutation
- Proteins