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 · 21h

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,

HealthDay on MSN4h

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 ...

GlobalData on MSN16h

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 ...

STAT18h

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

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 ...

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, ...

BioPharma Dive20h

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.

Buy Rating Affirmed for Viking Therapeutics on Promising VK0214 Trial Results and Strategic Focus on Obesity Program

Analyst Naz Rahman of Maxim Group reiterated a Buy rating on Viking Therapeutics (VKTX – Research Report), retaining the price target of ...

GlobalData on MSN13h

Bluebird bio’s Skysona led to seven cases of blood cancer in gene therapy trials

Whilst the events do not outline any new safety risks, the data gives a new understanding of the link between oncogenesis and ...

Business Wire2h

Poxel Announces Availability of the 2023 Universal Registration Document

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

Viking Therapeutics Reports Positive Data From Phase 1b Study Of VK0214 In X-ALD

(RTTNews) - Viking Therapeutics, Inc. (VKTX), Wednesday announced positive data from Phase 1b study of VK0214 in patients with X-linked adrenoleukodystrophy (X-ALD). X-ALD is a rare and often fatal ...

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