© 2021 MJH Life Sciences and Neurology Live. All rights reserved.
© 2021 MJH Life Sciences™ and Neurology Live. All rights reserved.
March 07, 2021
The 2-stage study is an open-label, 2-stage clinical trial designed to evaluate safety and dose-escalation (stage 1) and safety and efficacy (stage 2) of surgically delivered AXO-AAV-GM2.
January 05, 2021
A recent analysis of UnitedHealthcare insurance claims revealed increases of almost 200% for some services between 2001 and 2016.
December 23, 2020
PTC Therapeutics announced the initiation of its third study of 2020 investigating vatiquinone.
December 16, 2020
These data follow a recent announcement from Sio Gene Therapies that the first patient in the high-dose cohort has been dosed.
February 05, 2020
Interim analysis compared ataluren in a real-world setting from the STRIDE registry with a matched cohort; the final data from the STRIDE registry are expected in 2025.
July 03, 2019
The profound consequences of the results guide decisions about testing. Who should be tested? And, just as important, who should not?
June 25, 2019
In this podcast, Heidi Moawad, MD interviews Cesar Ochoa-Lubinoff, MD, MPH, FAAP, about recent encouraging clinical trial results in patients with Angelman syndrome.