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 Pharmaceuticals is launching a phase 1 trial of ISIS-DMPKRx, an experimental drug for type 1 myotonic muscular dystrophy
Update (June 11, 2014): This story has been updated with additional information about the phase 1 trial of ISIS-DMPKRx in healthy volunteers and the planned trial in people with type 1 MMD, as well as the availability of a fact sheet on this drug provided by Isis Pharmaceuticals...
Mice with a severe SMA-like disease that were injected with a synthetic "antisense" molecule developed bigger, more structurally sound muscles than untreated mice
A team of research scientists has found that mice with a disease resembling a severe form of spinal muscular atrophy (SMA) that were treated with a gene-modifying molecule produced more of a needed protein throughout their spinal cords; developed bigger, stronger muscles; and survived longer than expected.
Human testing has begun of ISIS-SOD1-Rx, a compound designed to block production of a toxic protein in people with the SOD1-related form of familial (inherited) ALS
Isis Pharmaceuticals of Carlsbad, Calif., has begun a phase 1 clinical trial of its experimental compound ISIS-SOD1-Rx in people with familial (inherited) ALS caused by toxic SOD1 protein molecules.