Orchard Therapeutics Announces Promising Early Neurocognitive Outcomes from Ongoing Proof-of-concept Study of OTL-201 in MPS-IIIA streetinsider.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from streetinsider.com Daily Mail and Mail on Sunday newspapers.
All patients achieved sustained engraftment and supraphysiological SGSH enzyme levels with median 1.5 years follow-up Four out of five patients demonstrated gain of cognitive skills in line.
Investegate announcements from F. Hoffmann-La Roche Ltd, Positive new data for Roche’s Evrysdi in largest trial ever undertaken in patients with previously-treated spinal muscular atrophy (SMA)