Spinal Muscular Atrophy

Ethical Challenges of Nusinersen: Considerations When Counseling on Treatment

Ethical Challenges of Nusinersen: Considerations When Counseling on Treatment

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Although regarded as a miracle drug to some, nusinersen comes with some troublesome strings attached.

First-in-Class Inhibitor Designated Orphan Drug for Spinal Muscular Atrophy

First-in-Class Inhibitor Designated Orphan Drug for Spinal Muscular Atrophy

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By inhibiting the activation of myostatin, SRK-015 may promote a clinically meaningful increase in muscle mass and strength.

Later-Onset Spinal Muscular Atrophy Benefits From Nusinersen

Later-Onset Spinal Muscular Atrophy Benefits From Nusinersen

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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 trial

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.

Fatty Liver Disease Common in Spinal-Bulbar Muscular Atrophy

Fatty Liver Disease Common in Spinal-Bulbar Muscular Atrophy

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Because of the liver's prominent role in drug metabolism, clinicians should monitor patients' hepatic function.

Nusinersen Improves Motor Function, Survival in Infants With Spinal Muscular Atrophy

Nusinersen Improves Motor Function, Survival in Infants With Spinal Muscular Atrophy

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Patients who received nusinersen had a greater likelihood of survival compared to those who received sham treatment.

FDA Approves First Treatment for Spinal Muscular Atrophy

FDA Approves First Treatment for Spinal Muscular Atrophy

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The US FDA has approved the first drug for the treatment of spinal muscular atrophy, a genetic disease that often results in premature death.

New Drug for Spinal Muscular Atrophy Shows Promise

New Drug for Spinal Muscular Atrophy Shows Promise

Despite the benefits observed, the improvements still do not represent normal muscle function.

Gene Therapy Stops, Stabilizes Spinal Muscular Atrophy in Animal Model

Gene Therapy Stops, Stabilizes Spinal Muscular Atrophy in Animal Model

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The study was the first to model the disease in a large animal with similar physiology to humans.

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