Victoria Johnson

August 16, 2022

The AAV1 gene therapy, PBFT02, is being assessed in a phase 1/2 trial called upliFT-D (NCT04747431), which is planned to take place over the course of 24 months in patients with frontotemporal dementia with granulin mutations.

August 12, 2022

The deaths, occurring shortly after treatment infusion, were because of acute liver injury, which is a known risk associated with the Novartis gene therapy.

October 04, 2021

Children with AADC receiving treatment developed motor function and cognitive skills that persisted through up to 10 years of follow-up.

October 01, 2021

Patients with Duchenne muscular dystrophy receiving the treatment had a decline in Mid-level Performance of Upper Limb scores in the phase 2 trial of the cell therapy.

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.

June 17, 2021

While the relationship between sleep and cognition needs to be further studied, sleep-based interventions may be a future approach to help prevent cognitive decline.

June 16, 2021

Santhera and ReveraGen announced findings of the phase 2b VISION DMD study of the investigational therapy.

June 15, 2021

Bayer provided an update on their phase 1 and 1b studies of dopaminergic neurons and GDNF gene therapy, respectively, for the treatment of PD.

June 12, 2021

Results from the START study of medication switching in narcolepsy were presented at the 2021 SLEEP Annual Meeting.

June 10, 2021

Data presented at SLEEP 2021 also showed the treatment was associated with significant weight loss and reduction in BMI compared to placebo.