A phase 1-2 trial of ISIS-DMPKRx, an experimental compound for the treatment of type 1 myotonic muscular dystrophy, is now underway in the U.S. in adults with this disorder who are 20 to 55 years old and meet other study criteria.
Weeklong Campaign to Sell Custom-Designed T-shirts
Inspired by the Strength of MDA Families and Supporters
CHICAGO, Nov. 24, 2014 — The Muscular Dystrophy Association and Sevenly.org, known for its seven-day, limited edition charity T-shirt campaigns, announced today they will join forces in the fight against muscle disease with the sale of five custom-designed T-shirts to benefit individuals and families served by MDA.
Update (Dec. 1, 2014):Isis has announced that its partner, Biogen Idec, plans to conduct a phase 2 trial of ISIS-SMNRx in up to 25 presymptomatic newborns genetically predisposed to develop SMA, as well as a phase 2 study of this drug in approximately 20 patients with infantile or childhood-onset SMA who do not meet the inclusion criteria for the current phase 3 studies. These two additional studies could begin in the first half of 2015.
PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, says it will soon open a trial to test the safety and tolerability of its investigational drug RG7800 in adults and children with spinal muscular atrophy (SMA). The announcement was made in a Nov. 19, 2014, press release.