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Small Molecule
Risdiplam for Spinal Muscular Atrophy
Recruiting3 awardsPhase 4
Strasburg, Pennsylvania
This trial is for SMA patients who have been treated with nusinersen and are now crossover to 36 months of risdiplam monotherapy. The trial will track the most informative outcomes from the nusinersen trial, while adding the Box and Block Test as an additional measure of upper limb endurance and function.
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