Isis Pharmaceuticals has announced the opening of a U.S. trial to evaluate the safety and tolerability of ISIS-DMPKRx in adults with type 1 myotonic dystrophy
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.
Isis Pharmaceuticals has opened a second phase 3 trial to test its "antisense" drug for spinal muscular atrophy in children ages 2 to 12
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...
Isis and Biogen Idec are collaborating to develop antisense drugs for neurologic disorders; a trial of ISIS-DMPKRx for MMD1 (DM1) is expected in 2014
Update (Oct. 16, 2013): Isis announced in an Oct.
Isis Pharmaceuticals has launched a trial to test multiple doses of its experimental antisense drug ISIS-SMNRx in 24 children with SMA at four US centers
Update (Nov. 22, 2013): In a Nov. 22, 2013, press release, Isis Pharmaceuticals announced it would investigate a higher dose of ISIS-SMNRx in children with SMA than originally planned. It will add a 12-milligram cohort to the ongoing phase 1b/2a study.