Genentech, a member of the Roche Group, today announced positive data from the pivotal Part 2 of the SUNFISH study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study met its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after

Dear SMA community, Today, we announced an AVXS-101 intrathecal study update. In the update, we shared that the United States Food & Drug Administration (FDA) placed a partial hold on clinical trials for intrathecal (IT) administration of AVXS-101. This partial hold by the FDA only impacts the IT formulation and does not impact the currently

The below statement is a press release from Novartis and AveXis. Novartis today announced the United States Food & Drug Administration (FDA) placed a partial hold on clinical trials for intrathecal administration of AVXS-101. The announcement follows an AveXis communication to health authorities and clinical trial investigators based on findings from a small, AveXis-initiated pre-clinical study in