Spinal Muscular Atrophy
Although regarded as a miracle drug to some, nusinersen comes with some troublesome strings attached.
By inhibiting the activation of myostatin, SRK-015 may promote a clinically meaningful increase in muscle mass and strength.
Improved motor function was observed among more participants in the nusinersen group compared with those in the control group.
Nusinersen improves motor function in children with later-onset spinal muscular atrophy: The CHERISH trialFebruary 15, 2018
Intrathecal injections of nusinersen in pediatric later-onset spinal muscular atrophy (SMA) patients were associated with increased clinical motor function scores compared to sham treated patients.
Because of the liver's prominent role in drug metabolism, clinicians should monitor patients' hepatic function.
Patients who received nusinersen had a greater likelihood of survival compared to those who received sham treatment.
The US FDA has approved the first drug for the treatment of spinal muscular atrophy, a genetic disease that often results in premature death.
Despite the benefits observed, the improvements still do not represent normal muscle function.
The study was the first to model the disease in a large animal with similar physiology to humans.
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