adrenoleukodystrophy news

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 · 1d

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, according to results from a pair of studies.

U.S. News & World Report · 1d

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,

GlobalData on MSN18h

Viking reports data from X-linked adrenoleukodystrophy trial

Viking Therapeutics has announced positive results from its Phase Ib clinical trial of VK0214, a thyroid hormone receptor ...

HealthDay on MSN6h

Hematologic Cancer Develops in Some Patients Receiving Eli-Cel

Hematologic cancer develops in some patients receiving lentiviral elivaldogene autotemcel (eli-cel) gene therapy for cerebral ...

Business Insider10h

Buy Rating Affirmed for Viking Therapeutics on Promising Clinical Trials and Strategic Focus on Metabolic Diseases

The company’s recent Phase 1b trial of VK-0214, an oral therapy for the treatment of X-linked adrenoleukodystrophy (X-ALD), demonstrated significant reductions in very long chain fatty acids (VLCFAs ...

STAT21h

A crack of light for Pfizer

Pfizer shared that the combination of its drugs Talzenna and Xtandi extended survival in men with metastatic ...

1don MSN

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, ...

1don MSN

Viking Therapeutics reports positive data from trial of treatment for rare metabolic disorder X-ALD

Viking Therapeutics Inc. on Wednesday reported positive data from an early-stage trial of a treatment for the rare, and often ...

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.