ATA-200 is a one-time gene therapy for the treatment of γ-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5), a serious and debilitating condition that affects children and ...
No disease-modifying treatment has proven to alter LGMD ... contributed to an equal number of deals. The Limb-Girdle Muscular Dystrophy: Competitive Landscape combines data from the Pharma ...
for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). FDA grants rare pediatric disease designation to incentivize the development of new treatments for serious and life ...