News Releases

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New Gene Therapy Trial for Severe Neuromuscular Disorder in Children

November 09, 2017

Lurie Children's participates in Phase 1/2 clinical trial of gene therapy for X-linked myotubular myopathy.
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Drug Improves Muscle Function and Survival in Spinal Muscular Atrophy

November 06, 2017

More than half of the babies with infantile-onset spinal muscular atrophy (SMA) who were treated with nusinersen (Spinraza) gained motor milestones, compared to none of the babies in the control group.
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