Pembrolizumab Shows Potential in Progressive Multifocal Leukoencephalopathy Patient Subsets
April 11th 2019Pembrolizumab resulted in a decrease in the detection of programmed cell death protein 1 on lymphocytes in both cerebrospinal fluid and peripheral blood, with 5 of the 8 patients displaying clinical improvement or stabilization.
Zogenix Receives Refusal to File Letter for Dravet Syndome Treatment ZX008
April 9th 2019The FDA cited 2 deficiencies in the NDA: certain nonclinical studies were not included to allow for assessment of chronic administration of fenfluramine; and an incorrect version of the clinical data was submitted.
Huntington Disease Gene Therapy AMT-130 Wins FDA Fast Track Designation
April 8th 2019The planned phase 1/2 trial of the recombinant AAV5 vector treatment, the first one-time administered AAV gene therapy to enter clinical testing for Huntington disease, is expected to begin dosing patients in the second half of 2019.
Pascal Sati, PhD: Challenges in Imaging for Multiple Sclerosis
April 8th 2019The senior preclinical and clinical imaging scientist at the National Institute of Neurological Disorders and Stroke gave a presentation on a volumetric segmented echo-planar-imaging (3D-EPI) sequence, which could be used to detect novel biomarkers such as the central vein sign rapidly.
Guidelines for Pediatric Migraine Clinical Trial Design Released
April 8th 2019The International Headache Society has issued a number of recommendations for the proper design of trials for the prevention of pediatric migraine, hoping to address the challenges which are unique to pediatric patient populations.