The CGTLive™ Neurology specialty topic page houses video interviews with key opinion leaders in the field of neurology about the latest relevant FDA actions, clinical guideline updates, and clinical trial findings related to cell therapies, gene therapies, and engineered and regenerative medicines developed for neurologic disorders and diseases. It also contains up-to-date clinical news coverage in the field of gene and cell therapy for central nervous system disorders.
October 4th 2024
Review top news and interview highlights from the week ending October 4, 2024.
Gene Therapy for CLN6 Batten Disease Shows Positive Interim Results
August 3rd 2019Interim data from the first 8 pediatric patients showed that the AAV-CLN6 gene therapy demonstrated a positive impact on motor and language function compared to a natural history dataset, as well as in comparison to in-study sibling pairs.
FDA Approves Gene Therapy With $2.1M Price Tag for Spinal Muscular Atrophy in Pediatric Patients
May 24th 2019AveXis—a Novartis company—announced that it will work with payers to implement 5-year outcomes-based agreements and novel pay-over-time options. The company also said it will offer a patient program to support affordability and access.
Can Striatal CaV1.3 Genetic Silencing Prevent Levodopa-Induced Dyskinesias in Parkinson?
May 17th 2019New early-stage data suggest that vector‐mediated gene silencing of striatal CaV1.3 expression may hold promise for preventing the induction of levodopa-induced dyskinesias in Parkinson disease.
FDA Grants Breakthrough Therapy Designation to Gene Therapy for Cerebral Adrenoleukodystrophy
June 3rd 2018The FDA has granted a Breakthrough Therapy designation to bluebird bio, Inc’s Lenti-D, a gene therapy for patients with cerebral adrenoleukodystrophy, an X-linked genetic disorder caused by a defect in the gene ABCD1.