Favorable outcomes with onasemnogene abeparvovec in UAE cohort

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May 01 / Springer Healthcare

Favorable outcomes with onasemnogene abeparvovec in UAE cohort

Neuroscience Spinal Muscular Atrophy EPNS-2022

Description

Vivek Mundada talks about an observational study of onasemnogene abeparvovec in a cohort of children with spinal muscular atrophy in the United Arab Emirates, highlighting that there were no complications despite a transient drop in platelets after gene therapy administration.

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