GlobalData on MSN18h
Viking reports data from X-linked adrenoleukodystrophy trialViking 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-CelHematologic cancer develops in some patients receiving lentiviral elivaldogene autotemcel (eli-cel) gene therapy for cerebral ...
Cerebral adrenoleukodystrophy (CALD) is a rare progressive, genetic brain disease that primarily presents in young boys, ...
Most boys treated with breakthrough gene therapy for a rare but deadly brain illness are faring well six years later, ...
Medpage Today on MSN1d
Gene Therapy for Rare Brain Disease Effective, but Comes With Blood Cancer RiskWhile the use of elivaldogene autotemcel (eli-cel; Skysona) gene therapy has demonstrated long-term efficacy in patients with ...
Seven out of 67 children who were given bluebird's (BLUE) gene therapy Skysona during clinical trials ended up developing ...
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), ...
Pfizer shared that the combination of its drugs Talzenna and Xtandi extended survival in men with metastatic ...
GlobalData on MSN15h
Bluebird bio’s Skysona led to seven cases of blood cancer in gene therapy trialsWhilst the events do not outline any new safety risks, the data gives a new understanding of the link between oncogenesis and ...
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