Study identifies miRNAs upregulated in SMA

Emanuela Abiusi outlines the results of a study showing that certain microRNAs are upregulated in muscle biopsy and serum samples taken from people with spinal muscular atrophy, and discusses the potential of these microRNAs as a target for SMN-independent therapies (4:46).

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).