Author name: Morgan Tuttle

Biogen and Ionis Pharmaceuticals recently provided an update on the clinical development of nusinersen (IONIS-SMNRx), which is currently being tested in Phase 3 clinical trials: Dear members of the SMA community, As we begin 2016, we wanted to provide an update on the nusinersen (IONIS-SMNRX) program to you and the many families around the world

Roche, along with partners PTC Therapeutics and the SMA Foundation, recently provided the following update on the development of RG7800 and on a new clinical trial planned for RG7916: “Dear SMA community, “The collaboration between Roche, PTC Therapeutics and the SMA Foundation would like to provide you with an update on the advance of our

AveXis has announced that they have completed enrollment in a Phase 1 study testing systemic delivery of gene therapy for SMA. The trial has enrolled a total of 15 patients who met enrollment criteria of diagnosis of SMA Type 1 before six months of age, with two copies of the SMN2 backup gene, as determined

Cytokinetics has opened enrollment for a Phase 2 study testing CK-2127107 in teens and adults with SMA type II, III or IV. We are excited to see the continued progress of this drug into the next phase of clinical trials, and particularly excited to see this trial focused on teens and adults. This latest trial