October 22nd 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 9th 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 5th 2021
LX1004 and APB-102 are currently in the early phases of research to treat CLN2 Batten disease and SOD1 amyotrophic lateral sclerosis, respectively.
May 13th 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 5th 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.
FDA Clears IND Application for SOD1-ALS Gene Therapy
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.
Treatment Disparities, Neurologic Complications Clarified by COVID-19 Neurological Research
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.
AXO-AAV-GM2 Administered to First Patient in GM2 Gangliosidosis Trial
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.
Out-of-Pocket Costs Steadily Increasing for Neurologic Care
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
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
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
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
The profound consequences of the results guide decisions about testing. Who should be tested? And, just as important, who should not?
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