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 ...

Columnist Patrick Moeschen, who lives with limb-girdle muscular dystrophy, explains why controlling what he can is so ...

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 ...

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 ...

(AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that AB-1003 (also known as LION-101) has received rare pediatric disease ...