"Our preliminary findings show that German children with SMA, despite significant physical disability, have surprisingly good subjective HRQoL." – Erik Landfeldt, MSc, PhD Previous studies have found ...
The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.
Biogen Inc. announced positive interim results from a Phase 1 study of salanersen (BIIB115/ION306), a novel antisense oligonucleotide for treating spinal muscular atrophy (SMA). The data show that ...
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