Report gives update on boys with rare brain disease 6 years after gene therapy
Most boys treated with breakthrough gene therapy for a rare but deadly brain illness are faring well six years later, two new ...
STAT20h
Sarepta’s gene therapy is fully approved, but its stock has tanked. Here’s why
Since the FDA cleared Sarepta Thereapeutics' gene therapy for nearly all people with Duchenne, its stock price has tanked.
Gene therapy shows long-term benefit for patients with a rare pediatric brain disease
Cerebral adrenoleukodystrophy (CALD) is a rare, progressive, genetic brain disease that primarily presents in young boys, ...
Gene Therapy Researcher's Patent Dispute Cleared to Proceed Against UPenn, Program Director
The court determined it wasn't required to dismiss the case pursuant to the defendants' statute of limitations argument, as ...
Johns Hopkins Newsletter2d
Current progress and innovation for gene therapy delivery systems
As of October 2024, there are more than thirty FDA-approved gene therapies, spanning a diverse array of diseases such as ...
Magnetically regulated gene therapy tech offers precise brain-circuit control
A new technology enables the control of specific brain circuits non-invasively with magnetic fields, according to a ...
BioPharma Dive22h
New data underline cancer risk of Bluebird therapy for brain disease
Seven young boys given Bluebird's Skysona later developed blood cancers, findings that could shape how doctors balance the gene therapy’s risks against its benefit.
BioSpace1d
Big Pharma’s Interest Grows in Evolving Cell and Gene Therapy Sector
Large pharmaceutical companies were out in force at this week’s 2024 Cell & Gene Meeting on the Mesa, as they look to expand ...
Topic: gene therapy
Despite a bumpy history, through work and trust, gene therapy may have a future. Treatments involve adding a new gene, or ...
News Medical on MSN1d
Novel gene therapy holds potential in treating patients with epilepsy
Researchers from the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) are working on a therapy that holds potential in treating patients with epilepsy, a neurological ...