Genetic Disorders

October 22, 2021

The trial provided Class IV evidence that VY-AADC01 and the associated surgical delivery procedure were well-tolerated in patients aged 40 to 70 years with moderately advanced Parkinson disease.

August 09, 2021

In addition to the uplifted phase 1 study, Novartis also initiated the phase 3 STEER study, which will evaluate OAV-101 in treatment-naive patients with SMA type 2 aged between 2 and 18 years old.

August 05, 2021

LX1004 and APB-102 are currently in the early phases of research to treat CLN2 Batten disease and SOD1 amyotrophic lateral sclerosis, respectively.

June 10, 2021

Expert neurologists and epileptologists share best practices for transitioning a patient with TSC from adolescence into adulthood.

June 10, 2021

Elizabeth Thiele, MD, PhD, and Peter Crino, MD, PhD, comment on unmet needs and challenges in treating tuberous sclerosis complex.

May 13, 2021

The safety profile of the SLC13A5 vector was “excellent” according to study authors, with no adverse effects on weight, general activity, or survival in knockout mice and wild type littermates.

May 05, 2021

In preclinical data, VY-HTT01 showed a robust and durable reduction of huntingtin mRNA and protein while distributed across core areas of Huntington disease pathology.

April 29, 2021

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.

April 19, 2021

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.

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.