Neurology

In observance of Rare Disease Day, held this year on February 29, catch up on some of the latest data updates from clinical trials for rare diseases.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The clinical research director of the UCSF Multiple Sclerosis Center discussed situations where CAR-T clinical trials may or may not be a good option for patients with various neurologic autoimmune diseases.

Juliane Gust, MD, PhD, an assistant professor of neurology at University of Washington, Seattle Children's, discussed a study that is currently underway via the NIH’s CARnation Consortium that will seek to address this gap in knowledge.

Review top news and interview highlights from the week ending February 23, 2024.

The physician from St. Jude Children’s Research Hospital discussed the importance of involvement from patients and families while researching the long-term impact of CAR-T.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The assistant professor of neurology at University of Washington, Seattle Children's discussed efforts to address a gap in knowledge in the long-term effects of CAR-T, if any, on children’s neurological development.

The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed how far the field has come since founding the organization/

The clinical research director of the UCSF Multiple Sclerosis Center also discussed the high expense of producing individualized therapies like CAR-T.

The executive director of global clinical development at Ultragenyx Pharmaceutical discussed the statistical findings she presented at the 2024 WORLDSymposium.

The Prescription Drug User Fee Act date for the FDA’s decision on the supplement has been set for June 21, 2024.

Review top news and interview highlights from the week ending February 16, 2024.

The clinical research director of the UCSF Multiple Sclerosis Center discussed the importance of rigorous clinical trial design for determining whether CAR-T will truly be of benefit in autoimmune disease.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The chief medical officer of Encoded Therapeutics discussed ETX101 gene therapy and other initiatives in the company’s overall Dravet program.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed what he views as the next horizon in the field of gene therapy.

The clinical research director of the UCSF Multiple Sclerosis Center discussed the current standard of care for B-cell driven autoimmune diseases and how CAR-T could address unmet needs.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed his research on a new application of efgartigimod alfa (Vyvgart).

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

Review top news and interview highlights from the week ending February 9, 2024.

The gene-edited cell therapy also showed a safety profile consistent with that previously seen in patients with early-onset MLD.

Among the 5 patients treated in the study, sustained supraphysiological expression of the disease-targeted gene, SGSH, was rapidly achieved in the leukocytes and the plasma.

Hematological recovery was achieved and maintained in all 4 patients in the study.

The chief scientific officer of CureDuchenne discussed how nonviral approaches may address issues including gene size and redosability.