Max Farmer was born healthy but then diagnosed with a rare and genetic condition that causes progressive muscle wasting when ...

Ionis’ pipeline of antisense drugs is beginning to produce results – a separate alliance with Biogen resulted in development and approval of Spinraza (nusinersen), approved for spinal muscular ...

Cost-effectiveness body NICE has rejected Biogen’s Spinraza (nusinersen) therapy for the rare genetic muscle wasting disease spinal muscular atrophy (SMA) because of its high cost – but is in ...

Injecting medicines into amniotic fluid may be a viable strategy for treating SMA before birth, according to a new study in ...

Blood creatinine-to-cystatin-c ratio, an indicator of muscle mass and strength, may be a treatment response biomarker of SMA, ...