Neurology

The Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed the role of organelle transport in neuromuscular diseases such as CMT.

The phase 3b SMART trial included 24 patients in total who each weighed from 8.5 kg to 21 kg at the time of treatment.

RGX-202 delivers a slightly larger form of microdystrophin than other gene therapies, including the CT domain of dystrophin.

The lead scientist at Percheron Therapeutics discussed how ATL1102 could target inflammation as a secondary cause of muscle damage in DMD.

The clinical research director of the UCSF Multiple Sclerosis Center discussed the difference between developing targeted therapies for well-understood diseases like SCD and less well-understood diseases like MS.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

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

Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

In observance of Rare Disease Day 2024, the vice president of public policy and advocacy at the Muscular Dystrophy Association discussed advocacy and policy progress regarding rare neuromuscular diseases.

BioRestorative has submitted an IND amendment to compare BRTX-100 to sham injection instead of saline injection.

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.