Isis Pharmaceuticals announced that it has
initiated a Phase 1 study of ISIS-SMNRx in patients with spinal muscular
atrophy (SMA). SMA is a severe motor-neuron disease that is the leading
genetic cause of infant mortality. Isis is developing ISIS-SMNRx as a
potential treatment for all Types of SMA.
The Phase 1 study of ISIS-SMNRx is a single-dose, dose-escalation study
designed to assess the safety, tolerability and pharmacokinetic profile
of the drug in children with SMA between the ages of 2-14 who are
medically stable. In this study, ISIS-SMNRx will be administered
intrathecally as a single injection directly into the spinal fluid.
The study is estimated to be completed by October 2012.
Columbia University Medical Center is already recruiting for the study, the contact is Jacqueline Montes.
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