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PHENO SMART
Characterisation of new phenotypes in patients with type 1, 2 and 3 infantile or juvenile spinal muscular atrophy treated with therapies that restore SMN protein expression.
Support our Foundation of Myology project
Characterisation of new phenotypes in patients with type 1, 2 and 3 infantile or juvenile spinal muscular atrophy treated with therapies that restore SMN protein expression.