A gene therapy for spinal muscular atrophy (SMA) patients is making waves. Novartis (NYSE:NVS) revealed in a press release new long-term data highlighting the durability of Zolgensma (onasemnogene abeparvovec) up to 7.5 years after a single treatment. The data comes from two long-term follow-up studies, LT-001 and LT-002, which examined a range of patient populations…
Roche’s Evrysdi wins FDA approval in newborns with spinal muscular atrophy
Roche (SIX:RO, ROG; OTCQX:RHHBY) has announced that the FDA has approved a label extension to allow the use of Evrysdi (risdiplam) in babies younger than two months old with spinal muscular atrophy (SMA). SMA is a genetic disease associated with progressive muscle wasting and weakness that affects roughly 1 in 11,000 babies. The drug first…
Biogen plays up spinal muscular atrophy pipeline
As Biogen Inc. (Nasdaq: BIIB) continues to struggle to capitalize on the Alzheimer’s drug Aduhelm (aducanumab), the company is highlighting new data from its Spinraza (nusinersen) and spinal muscular atrophy (SMA) research program. First approved in 2016, Spinraza is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated to treat spinal muscular atrophy (SMA). At the…
Scholar Rock sees stock soar after announcing positive SMA results
A monoclonal antibody that blocks the activation of the skeletal muscle protein myostatin could be novel muscle-directed therapy for spinal muscular atrophy patients. Cambridge, Mass.–based Scholar Rock (NSDQ:SRRK), which is developing the SRK-015 investigational antibody, saw its shares more than double to $30.02 apiece yesterday. SRK-015 could lead to the formation of a new treatment…