Great News! A phase 1b trial of a drug designed to increase utrophin protein levels in Duchenne MD-affected muscles, appears safe and may have muscle-protective effects.


Article Highlights:

  • SMT C1100, an experimental drug designed to treat Duchenne and possibly Becker MD by raising utrophin protein levels in mature muscles, has shown encouraging results in a phase 1b trial; a new trial is planned for later in 2014.
  • The drug is being developed and tested by Summit PLC, an Oxford, United Kingdom, biotechnology company, with support from MDA.
  • The phase 1b trial in 12 boys with DMD showed SMT C1100 was safe and well-tolerated and that short-term administration of this oral drug was associated with a reduction in serum CK level, a positive indicator of muscle health.

See Full Story Here: