Call for screening for spinal muscular atrophy

Better results treating before symptom onset

New disease-modifying treatment available for spinal muscular atrophy can greatly improve outcomes if delivered before symptoms appear, neurologists say in calling for newborn screening for the condition.

Dr Hugo Sampaio and Dr Michelle Farrar, from Sydney Children’s Hospital, and newborn screening expert Professor Bridget Wilcken, from the Sydney children’s and Westmead children’s hospitals have co-authored an MJA Perspective article making a case for screening.

Nusinersen (Spinraza, Biogen Australia) was registered by the TGA in November for the rare autosomal

Latest