Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome
NEJM 385:1929-1940, Gentner, B.,et al, 2021
Nusinersen Versus Sham Control in Later-Onset Spinal Muscular Atrophy
NEJM 378:625-635, Mercuri, E.,et al, 2018
EMA401, An Orally Administered Highly Selective Angiotensin II Type 2 Receptor Antagonist, as a Novel Treatment for Postherpetic Neuralgia: A Randomised, Double-Blind, Placebo-Controlled Phase 2 Clinical Trial
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