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 ...

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, ...

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 ...

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 ...

Business Wire1h

Poxel Announces Availability of the 2023 Universal Registration Document

POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for ...

Medscape6d

Adrenoleukodystrophy: Recent Advances in Treatment and Disease Etiology

X-linked adrenoleukodystrophy (X-ALD) is a debilitating disease caused by mutations in the ABCD1 gene. Approximately 30% of affected boys develop the progressive ...

Nature1mon

Gene redundancy and pharmacological gene therapy: Implications for X-linked adrenoleukodystrophy

X-linked adrenoleukodystrophy (X-ALD) is a peroxisomal disorder with impaired very-long-chain fatty acid metabolism. The X-ALD gene encodes a peroxisomal membrane protein (ALDP) that is part of a ...

San Francisco State Golden Gate Xpress11d

Student parents struggle to find their place on campus

Esteban Carrasco is constantly running. From his University Park South dorm, he runs to classes, different on-campus ...

14hon MSN

7 children given bluebird's Skysona developed blood cancer: study

Seven out of 67 children who were given bluebird bio’s (NASDAQ:BLUE) gene therapy Skysona during clinical trials ended up ...

Results that may be inaccessible to you are currently showing.

Hide inaccessible results

Related topics