Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline
Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014.
PTC is moving forward with ataluren
Hansell Stedman, a surgeon who had two brothers with Duchenne muscular dystrophy, aims to translate lessons from crush injuries into improvements in gene therapy
You don’t have to look far to figure out how Hansell Stedman got interested in Duchenne muscular dystrophy (DMD). His older brother, Holt, and younger brother, Roland, had the disease.
MDA-supported biotechnology company Summit PLC and the University of Oxford will collaborate on development of utrophin modulators to treat Duchenne/Becker MD
MDA-supported biotechnology company Summit PLC has entered into a collaboration with the University of Oxford (United Kingdom) for continued development of utrophin modulators to treat Duchenne muscular dystrophy (DMD) and possibly Becker muscular dystrophy (BMD).
Summit PLC, an MDA-supported biotechnology company in the UK, has received approval to test its experimental DMD drug SMT C1100 in boys with this disorder
Update (Feb. 2, 2014): Results of this trial are expected during the second quarter of 2014. For more information about this United Kingdom-based trial, see Phase 1b Study of SMT C1100 in Subjects with Duchenne Muscular Dystrophy; or enter NCT02056808 into the search box at ClinicalTrials.gov.
The drug SMT C1100 is still experimental, but an MDA-supported company has received two US patents that provide commercial protection for its development
Summit PLC, a biotechnology company based in Abingdon, United Kingdom, has announced it now has two U.S. patents for its experimental drug SMT C1100 for Duchenne muscular dystrophy (DMD).
Mice with a disorder similar to Duchenne muscular dystrophy showed improved muscle strength and structure following treatment with genetically corrected cells derived from their own skin cells
A therapeutic strategy that combines gene therapy and stem cell transplantation has shown encouraging results in mice with a disorder mimicking Duchenne muscular dystrophy (DMD).
A follistatin gene therapy compound gains orphan drug status; potential improvements are reported for utrophin-based therapies, exon skipping and stem cell transplantation
Update (Jan. 23, 2013): The "Building better utrophin" section was updated to reflect the availability of a Jan. 22, 2013, press release from the University of Missouri.
Below is a wrap-up of recent research news about the development of therapies for Duchenne, Becker and limb-girdle muscular dystrophies.