Genpharm's perspective on the rare disease landscape in the MENA region.

Biologic drugs have transformed the therapeutics landscape in numerous areas of healthcare. 

The muscular dystrophies are a group of inherited disorders described by progressive muscle degeneration and weakness.

A neuromuscular disease (NMD) is a disorder that affects the peripheral nervous system.

SBMA is a genetic disorder which affects the part of the nervous system that controls voluntary muscle.