IND-enabling studies support further evaluation of SMARD1/CMT2S gene therapy

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Mar 16 / Springer Healthcare

IND-enabling studies support further evaluation of SMARD1/CMT2S gene therapy

Neuroscience Spinal Muscular Atrophy MDA-2022

Description

Kathrin Meyer talks about IND-enabling efficacy and safety studies of IGHMBP2 gene therapy in animal models of spinal muscular atrophy with respiratory distress type 1/Charcot-Marie-Tooth disease type 2S, the results of which support progression into phase 1 and 2 clinical trials.

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