Review top news and interview highlights from the week ending March 24, 2023.
Welcome to CGTLive’s Weekly Rewind! This week’s coverage highlights the recent 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference the team covered live on site, through expert interviews and session coverage.
The patient was treated at the new fifth dose level and had concomitant metapneumovirus infection.
The co-founder and chief executive officer of MyoGene Bio spoke about the company's gene editing therapy for Duchenne muscular dystrophy at MDA’s 2023 conference.
Presented at MDA 2023, the gene therapy (also known as delandistrogene moxeparvovec) showed significant improvements on NSAA total score, 10-meter walk/run scores, and time to rise scores relative to an external control cohort.
The chief medical officer of QurAlis discussed the antisense oligonucleotide therapy QRL-201.
Data from the phase 4 RESPOND trial were presented at the 2023 MDA Conference.
Choosing the Right Patient Populations for CAR-T Clinical Trials in Neurologic Autoimmune Disease
March 21st 2024Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed considerations for evaluating CAR-T in patients most likely to obtain meaningful benefit.