Facioscapulohumeral muscular dystrophy (FSHD) is a skeletal muscular dystrophy characterized by DNA hypomethylation of D4Z4 repeat units of a macrosatellite array found at the distal end of chromosome region 4q35, which causes a myotoxic expression of DUX4. Researchers from Epic-Bio presented the discovery of EPI-321, a novel gene therapy candidate for the treatment of FSHD.

Calcium-dependent protease calpain-2 (CAPN2) has been previously proposed as a critical effector of axonal degeneration, which is a key early...

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by failure of motor neurons that lead to paralysis. To date, no treatment exists for ALS that focuses on improving neuromuscular transmission, which would improve quality of life for ALS patients.

If you believe the theme of the World Dementia Council (WDC) meeting in London this week, dementia is “in a new era,” where it will be possible to prevent, diagnose and treat neurodegenerative disease. That is not the case for most people living with dementia today, but the approval of the first disease-modifying drugs and the imminent arrival of new blood-based biomarkers is “a big moment,” Lenny Shallcross, executive director of WDC told the meeting on Mar. 20.