Author Archives: Lindsey Shapiro PhD

New Gene Therapy Shows Potential for Safety, Efficacy in Mouse Model

A second-generation investigational gene therapy for spinal muscular atrophy (SMA) showed greater safety and efficacy than an earlier version, and it may be more effective than the currently approved gene therapy for SMA, a study in a disease mouse model reported. The study was conducted by scientists with CANbridge…

2 UK Nonprofits Fund PhD Student to Study SMA Mechanisms, Treatments

A couple of nonprofits in the U.K. will jointly sponsor a PhD student’s research into the mechanisms underlying nerve degeneration and potential treatment approaches for spinal muscular atrophy (SMA). Totaling £113,022 (nearly $150,000) and funded evenly by Spinal Muscular Atrophy UK (SMA UK) and Muscular Dystrophy UK (MDUK),…