The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...
A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in children and adolescents with spinal muscular atrophy, according to a study ...
Spinal muscular atrophy is a rare genetic disorder causing progressive muscle weakness, now in focus after Jesy Nelson shared her twins' diagnosis.
A 5-year-old British boy with the rare disease spinal muscular atrophy (SMA) has miraculously begun walking four years after receiving the world’s most expensive gene therapy. On the 31st, the BBC ...