Clinical trial highlights

We present updated results from ASPIRO (NCT03199469), investigating gene replacement therapy with AT132 for XLMTM patients. XLMTM, an ultra-rare, life-threatening myopathy caused by mutations in the MTM1 gene, leads to impaired neuromuscular and respiratory function, and early death. Patients enrolled required ventilator support and had no clinically significant underlying liver disease at baseline, defined as>5x ULN ALT or AST, or hepatic peliosis by imaging. As of Jul2020, efficacy data were analysed for 16 patients (n=6, 1  × 1014 vg/kg (age 0.8–4.1 years at dosing); n=10, 3 × 1014 vg/kg (age 1.3–6.8 years at dosing).

Source: Neuromuscular Disorders - September 19, 2021 Category: Neurology Authors: P. Shieh, N. Kuntz, J. Dowling, W. M üller-Felber, A. Blaschek, C. Bönnemann, R. Foley, D. Saade, A. Seferian, L. Servais, M. Lawlor, M. Noursalehi, S. Prasad, S. Rico, W. Miller Source Type: research