A new DRUG TRIALS SNAPSHOT FOR SPINRAZA is now available.
SPINRAZA is a drug for the treatment of spinal muscular atrophy (SMA).
Spinal muscular atrophy is a rare genetic disease that affects mostly children and young adults. It is caused by a low level of protein in the motor neuron that are responsible for normal muscle functioning. The lack of protein causes motor neuron loss, progressive muscle weakness and may lead to premature death from respiratory failure.
Drug Trials Snapshots provide information about who participated in clinical trials that supported the FDA approval of new drugs. The information provided in these Snapshots also highlights whether there were any differences in the benefits and side effects among sex, race and age groups.
See more Drug Trial Snapshots or contact us with questions at Snapshots@fda.hhs.gov.
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