The experimental antisense drug will be tested in infants with a genetic spinal muscular atrophy diagnosis who are to date without symptoms
posted on March 16, 2015 - 11:03am
Biogen Idec has announced plans to run a phase 2 clinical study of the experimental drug ISIS-SMNRx  in infants with spinal muscular atrophy (SMA)who are less than 6 weeks old and have a genetic diagnosis of the disorder but are not yet showing symptoms. 

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

posted on December 22, 2014 - 5:12pm
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

posted on November 21, 2014 - 10:19am
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 Pharmaceuticals is testing its experimental, antisense-based drug in a phase 3 trial in SMA-affected babies age 7 months or less

posted on August 1, 2014 - 10:24am
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

posted on September 10, 2013 - 4:55pm
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

posted on May 7, 2013 - 12:15pm
  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

posted on March 21, 2013 - 11:31am
An experimental drug designed to treat the underlying molecular defect in spinal muscular atrophy (SMA) has shown encouraging results in a phase 1 trial.

New imaging techniques illuminate muscle and nerve, speeding research and aiding clinical trials

posted on July 1, 2012 - 3:09pm
QUEST Vol. 19, No. 3
Researchers are very good at understanding what is going on with things they can’t see directly, whether it be the membranes of muscle cells or the motor neurons of the spinal cord. But what is true for the rest of us is equally true for scientists: Seeing something is often the quickest and most direct way to understand it.