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.

Nurexone Biologic Inc. has released further results from a preclinical study of Exopten demonstrating effective treatment of 75% of paralyzed rats suffering from spinal cord injuries.

The neuron-specific K+/Cl- co-transporter KCC2 is involved in the shift from depolarizing to hyperpolarizing γ-aminobutyric acid receptor (GABAAR) currents and subsequent synaptic inhibition.