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

Life scientist researching in the laboratory.

Kathrin Meyer talks about IND-enabling efficacy and safety studies of IGHMBP2 gene therapy in animal models spanning the disease spectrum 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 (2:46).

JNK inhibition shows promise for SMA

Laxman Gangwani outlines the results of a preclinical study showing that JNK inhibition is a promising therapeutic strategy for spinal muscular atrophy, and discusses how this approach might impact the treatment landscape if shown to be beneficial in clinical trials (4:22).

Support for onasemnogene abeparvovec in older SMA patients

Laurent Servais discusses real-world research suggesting that onasemnogene abeparvovec is beneficial in spinal muscular atrophy patients aged 6 months and older, supporting the results of clinical trials involving younger patients (4:13).