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
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).
SMT C1100, which increases levels of the protein utrophin, reached adequate blood levels in healthy volunteers; trials in Duchenne MD may be next
The experimental drug SMT C1100 is designed to increase utrophin production as a treatment for Duchenne MD
MDA has awarded $750,000 to Summit Corp. to test a new formulation of its utrophin booster SMT C1100 for Duchenne muscular dystrophy
MDA has awarded $750,000 to Summit Corporation PLC for development and testing of SMT C1100, the company's experimental drug for treatment of Duchenne muscular dystrophy (DMD). Summit is an Oxford, United Kingdom, drug discovery company.
The award was made through MDA's Venture Philanthropy (MVP) arm, a part of MDA's translational research program.
New MDA clinical research network to target ALS, DMD