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 ...
Medpage Today on MSN1d
Gene Therapy for Rare Brain Disease Effective, but Comes With Blood Cancer Risk
While the use of elivaldogene autotemcel (eli-cel; Skysona) gene therapy has demonstrated long-term efficacy in patients with ...
Medscape6d
In Gene Therapy for RP, LCA, the Earlier, the Better
A real-world study of an FDA-approved gene therapy for inherited retinal disease shows younger patients gain more visual ...
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 ...
Novel gene therapy shows promise in targeting a mutation linked to epilepsy
Researchers from the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) are working on a ...
STAT18h
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 offers hope for children with cerebral adrenoleukodystrophy
Cerebral adrenoleukodystrophy (CALD) is a rare progressive, genetic brain disease that primarily presents in young boys, ...
FierceBiotech2d
Purespring bottles up $105M series B to bring kidney disease gene therapy to the clinic
London-based biotech Purespring Therapeutics plans to bring their kidney-tweaking gene therapy to the clinic with the help of ...
sciencex1d
Improving access to cell and gene therapy in pediatrics
Science X is a network of high quality websites with most complete and comprehensive daily coverage of the full sweep of ...
STAT12h
7 children developed blood cancer after Bluebird Bio gene therapy for rare neurological disease
A Bluebird Bio gene therapy has helped children with a devastating neurological disorder, but seven have developed blood ...
Amwins Group Benefits Expands Gene Therapy Solutions Program
Today, Amwins Group Benefits announced the expansion of its Gene Therapy Solutions (GTS) program. Now covering 15 revolutionary gene and cell therapies, the proprietary program helps employers ...