Genetic Disorders

Apic Bio plans to initiate a phase 1/2 clinical trial in late 2021 or early 2022 as a multi-center, 3-part study to evaluate APB-102 in patients with SOD1-ALS mutations.

The 2021 AAN Meeting Hot Topics plenary focused on neurological facets of COVID-19, and featured insight from Anthony Fauci, MD, of NIAID, and Walter Koroshetz, MD, of NINDS, among others.

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.

A recent analysis of UnitedHealthcare insurance claims revealed increases of almost 200% for some services between 2001 and 2016.

PTC Therapeutics announced the initiation of its third study of 2020 investigating vatiquinone.

These data follow a recent announcement from Sio Gene Therapies that the first patient in the high-dose cohort has been dosed.

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.

The profound consequences of the results guide decisions about testing. Who should be tested? And, just as important, who should not?