Isis Pharmaceuticals is testing its experimental, antisense-based drug in a phase 3 trial in SMA-affected babies age 7 months or less
California-based Isis Pharmaceuticals has announced the opening of a phase 3 clinical trial of its experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA), a muscle-weakening disease that results from loss of nerve cells in the spinal cord.
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 phase 2 trial to test its experimental antisense drug ISIS-SMNRx in infants with spinal muscular atrophy
Update (Jan. 14, 2014): Contact information for Isis Pharmaceuticals has been changed.
Update (Nov. 4, 2013): All centers are now recruiting participants.
Encouraging results were seen in an early-stage trial of ISIS-SMNRx, a drug designed to increase levels of a deficient protein in spinal muscular atrophy
An experimental drug designed to treat the underlying molecular defect in spinal muscular atrophy (SMA) has shown encouraging results in a phase 1 trial.
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.