Clinical Trials
AveXis Receives Orphan Drug Designation
AveXis has received Orphan Drug Designation from the FDA for their gene therapy program, called chariSMA. Orphan Drug Designation is granted by the FDA to […]
Read More ›Isis Begins Recruiting for Phase III Trial
Isis is currently recruiting participants for Endear, a pivotal Phase III study evaluating ISIS-SMNRx in infants with spinal muscular atrophy (SMA). The goal of this […]
Read More ›Spinal Muscular Atrophy Biomarker Study Reaches Enrollment Goals
We’re excited to announce that enrollment for the NeuroNEXT biomarker study is now fully filled. This means that this critical study can continue to progress […]
Read More ›Cure SMA and PPMD Invite Spinal Muscular Atrophy Parents to Take Part in Two Clinical Trial Surveys
Cure SMA and Parent Project Muscular Dystrophy (PPMD) are conducting two different surveys about clinical trial participation. The goal is to find out more about […]
Read More ›Spinal Muscular Atrophy Mice Model Results Published in the Journal Science
On August 7, the journal Science published results of a preclinical study demonstrating that treatment with orally available RNA splicing modifiers of the SMN2 gene […]
Read More ›Isis Begins Phase III Clinical Trial
On August 1, Isis Pharmaceuticals, Inc. announced today the initiation of a pivotal Phase III study evaluating ISIS-SMNRx in infants with spinal muscular atrophy (SMA). […]
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