On June 11, Isis Pharmaceuticals, Inc. released an update on its ongoing open-label Phase 2 clinical study of ISIS-SMNRx in infants with Type I spinal muscular atrophy (SMA).  The data showed that infants treated with ISIS-SMNRx have shown continued increases in median event-free survival, positive results in muscle function tests, and attainment of important developmental milestones.  The safety and tolerability profile of ISIS-SMNRx also continues to support further development.

ISIS-SMNRx is an experimental drug being developed by Biogen Idec and Isis Pharmaceuticals and is designed to raise levels of the SMN protein, which is deficient in the cells of SMA patients.  This Phase 2 trial is testing the drug in 20 infants diagnosed with SMA who were seven months or younger at the time they received the drug.

“The trial results are extremely exciting for our SMA community. SMA is a devastating disease for which there are currently no treatments. These positive results from ISIS gives us hope that we will soon have an effective therapy to change the lives of these infants and their families.” said Amanda M. Haidet-Phillips, Ph.D., MDA Scientific Program Officer. “These are very encouraging Phase 2 results, and we are hopeful that the Phase 3 double-blind, randomized study will have positive outcomes. MDA-supported laboratory research contributed to the development of ISIS-SMNRx, and this news shows our commitment to pursuing lifesaving discoveries.”

About MDA

The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to saving and improving the lives of people with muscle disease, including muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research to find treatments and cures; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.

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