Alnylam Pharmaceuticals reported initial Phase 1b findings showing brezosiran reduced cerebrospinal fluid mutant huntingtin levels by a mean of 60% to 64% at 3 months, with plans for a pivotal study in 2027.
|Slideshows|April 24, 2017
Parkinson Disease: Genetic Findings
Author(s)Samantha J. Peterson
Research at AAN 2017 highlights research of genes that may help predict the likelihood of developing Parkinson disease and progression of the disease.
Advertisement
Studies from AAN 2017 presented research examining lysosomal storage disorder (LSD) genes associated with risk of Parkinson disease, questioning whether specific mutations of GBA gene impact progression of the disease, and tackling the challenge of utilizing whole-exome sequencing to identify genes linked to Parkinson disease risk in a large cohort of unrelated subjects.From materials presented at AAN Annual Meeting, Boston, MA. Best of: Parkinson Disease session. April 23, 2017.
Advertisement
Related to this article
The FDA accepted uniQure's biologics license application for ifezuntirgene inilparvovec and granted it Priority Review, setting a target action date of May 1, 2027.
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 23, 2026.
Advertisement
Advertisement
Trending on NeurologyLive - Clinical Neurology News and Neurology Expert Insights
1
FDA Grants Priority Review to Huntington Disease Gene Therapy BLA
2
Zola-Cel and Treatment-Free Remission: What the BREAKFREE-2 Data Show
3
Examining Trontinemab in Early Alzheimer Disease: The Phase 3 TRONTIER 1 and 2 Trials
4
Understanding Cemdisiran's Mechanism of Action in Myasthenia Gravis
5