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Genetic Disorders
Out-of-Pocket Costs Steadily Increasing for Neurologic Care
January 05, 2021
Article
A recent analysis of UnitedHealthcare insurance claims revealed increases of almost 200% for some services between 2001 and 2016.
MOVE-FA Study Initiated for Friedriech’s Ataxia in Children and Young Adults
December 23, 2020
Article
PTC Therapeutics announced the initiation of its third study of 2020 investigating vatiquinone.
Initial Results Prove Positive for Low-Dose AXO-AAV-GM1 in GM1 Gangliosidosis
December 16, 2020
Article
These data follow a recent announcement from Sio Gene Therapies that the first patient in the high-dose cohort has been dosed.
Ataluren Extends Ability to Walk in Patients With Duchenne Muscular Dystrophy
February 05, 2020
Article
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.
Genetic Testing for Huntington Disease
July 03, 2019
Article
The profound consequences of the results guide decisions about testing. Who should be tested? And, just as important, who should not?
Phase 2 Trial for Angelman Syndrome Underway
June 25, 2019
Podcast
In this podcast, Heidi Moawad, MD interviews Cesar Ochoa-Lubinoff, MD, MPH, FAAP, about recent encouraging clinical trial results in patients with Angelman syndrome.