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gene therapy, Brain Disease

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 reports find.

Medpage Today on MSN · 2d

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 cerebral adrenoleukodystrophy (CALD), that benefit comes with a risk of developing hematologic cancers,

U.S. News & World Report · 20h

Boys Treated With Gene Therapy for Rare Brain Disease Doing Well 6 Years Later

Most boys treated with breakthrough gene therapy for a rare but deadly brain illness are faring well six years later, two new reports find. The 77 boys were treated for cerebral adrenoleukodystrophy (CALD),

Science Daily · 1d

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, causing loss of neurological function and ultimately leading to early death. Researchers have shown that six years after treatment with the first gene therapy approved for CALD,

STAT10h

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 ...

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 ...

12hon MSN

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 ...

STAT16h

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.

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 ...

BioPharma Dive19h

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.

Medical News Today9d

Is gene therapy the next big step in vision loss treatment?

A number of recent clinical trials have suggested that gene therapy could help treat diseases of the eye that cause vision ...

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 ...

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 ...

1don MSN

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 ...

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