Gene Therapy for AADC Deficiency Receives Positive CHMP Opinion

The positive opinion was based on findings from studies conducted in Taiwan.

CGTLive’s Weekly Rewind – May 20, 2022

Review top news and interview highlights from the week ending May 20, 2022.

DMD Gene Therapy Shows Continued Microdystrophin Expression

Motor and pulmonary function have stabilized or improved in participants treated with SGT-001.