Gene Therapy

November 22nd 2023

A new gene therapy administered directly to the brain aims to preserve dopamine neurotransmission in patients with multiple system atrophy.

November 14th 2023

Payers must be early adopters of managing genetic testing through DEX Z-Codes, combined with science-based policies and policy adherence and claims editing technology.

October 31st 2023

Sarepta Therapeutics plans to submit an application for full approval of Elevidys to treat all ages of patients with Duchenne muscular dystrophy. Company officials said the FDA is open to evaluating the application based on the totality of the evidence.

October 30th 2023

Many carriers reported feelings of concern, anxiousness, and guilt for passing the X-linked inherited retinal disease to their children—and 78% of respondents in a new study believe that carriers should have access to gene therapy options.