The U.S. Food and Drug Administration approved the six-minute walk test (6MWT) as an acceptable primary endpoint, or goal, for a potential registration program of the investigational treatment reldesemtiv for spinal muscular ... Read more
Infants with spinal muscular atrophy (SMA) continue to show better motor control and strength the longer they receive Spinraza (nusinersen), concludes a new and long-term analysis of final data from a ... Read more
The gene therapy developer AveXis will start a pivotal clinical trial of AVXS-101 for people with SMA type 1.
U.S. Food and Drug Administration officials agreed to the trial after AveXis submitted information the ... Read more
The U.S. Food and Drug Administration (FDA) has granted Cytokinetics’ investigational spinal muscular atrophy (SMA) treatment CK-2127107 Orphan Drug Designation. That gives it seven years of U.S. marketing exclusivity, tax ... Read more
Cytokinetics has begun enrolling a second group of patients for a Phase 2 clinical trial of CK-2127107 as a treatment for spinal muscular atrophy (SMA). The announcement follows an independent Data ... Read more
Nusinersen, an investigational drug for the treatment of spinal muscular atrophy (SMA) is acceptably safe and well tolerated, and shows encouraging clinical efficacy according to a study published in ... Read more
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