Bruce 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 the importance of further research into the root cause of MS and other autoimmune diseases.
The associate professor of pediatrics at Emory University also discussed the need to empower patients and families to make their own treatment decisions.
Avidity is currently enrolling in the phase 3 HARBOR trial, which it expects to initiate in the second quarter of 2024.
John Ligon, MD, an assistant professor in the department of pediatrics at the University of Florida College of Medicine, discussed his view of the main priorities for research in this area.
The endowed chair in cellular and molecular medicine at Boston Children’s Hospital discussed the rapid advancements in RNA-based treatments in the past 2 decades and potential advancements that remain on the horizon.
Bruce 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.
Updated data from the RESPOND study evaluating Spinraza in patients with SMA after onasemnogene abeparvovec were presented.
John Ligon, MD, an assistant professor in the department of pediatrics at the University of Florida College of Medicine, discussed his team’s early findings on CAR-T and fertility from CIBMTR centers.
The associate professor of clinical pediatrics at Cincinnati Children's discussed the latest data from the phase 1/2 STAAR study of isaralgagene civaparvovec.
Bruce 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 the potential impact of CAR-T therapy on the lives of patients with autoimmune disease.
Sandra P. Reyna, MD, the chief scientific advisor and head of global medical engagement for SMA at Novartis, discussed data from the phase 3b SMART clinical trial (NCT04851873) that were presented at MDA's 2024 conference.
The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed takeaways from a session on rare disease gene therapy at MDA.
The lead scientist at Percheron Therapeutics discussed the design of the study assessing proteomics of ATL1102.
Bruce 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 the need for randomized-control trials for CAR-T in lupus and other autoimmune diseases.
The Assistant Professor of Medicine at Weill Cornell Medical College discussed Orca-T cell therapy and next steps assessing it.
The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed his research career with muscular dystrophies.
The assistant attending physician and bone marrow transplant specialist at Memorial Sloan Kettering Cancer Center discussed updated data on GDA-201 plus rituximab.
The assistant professor in the department of pediatrics at the University of Florida College of Medicine discussed also discussed the latest results announced from the phase 2 portion of the PRISM study.
The assistant professor in the department of pediatrics at the University of Florida College of Medicine discussed also discussed the need to set standard guidelines regarding potential fertility issues for CAR-T treatment.
Bruce 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 the hypothesis behind research on the use of CAR-T for multiple sclerosis.
The professor of microbiology, immunology, and molecular genetics at UCLA discussed how a better understanding of treatment impact on a cellular level could help improve future gene therapy approaches.
The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed initiatives including the BGTC and the platform vector gene therapy project.
Peter A. Merkel, MD, MPH, the chief of the Division of Rheumatology and a professor of medicine and professor of epidemiology at Penn Medicine, discussed the significant challenges in this field, but also expressed optimism for the future.
The Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed talks from a session on dysfunction and trafficking at the 2024 MDA Conference.
The lead of preclinical research at Amylyx Pharmaceuticals discussed the literature supporting AMX0114’s target and its upcoming trial design.
The postdoctoral scholar at University of California – Irvine discussed his research looking at TDP-43 and alternative polyadenylation in neuronal cells.
The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed investigations into liver toxicity and deaths after AT132 gene therapy.
The data comes from 7 patients treated in the phase 1/2 IGNITE-DMD study (NCT03368742).
The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed initiatives including the BGTC and the platform vector gene therapy project.
The real-world data, which includes patients who received Zolgensma before and after tracheostomy, comes from the noninterventional RESTORE registry (NCT04174157).
