Positive news on bluebird bio's study of Lenti-D for boys with CALD
US biotech bluebird bio (Nasdaq: BLUE) has announced encouraging data from a Phase II/III study of the impact of its Lenti-D product candidate in pediatric patients with cerebral adrenoleukodystrophy (CALD).
Also known as Lorenzo’s Oil disease, CALD is a rare and fatal, X-linked, inherited, neurodegenerative disease that primarily affects young boys. The disease involves a progressive destruction of myelin, the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control.
Lenti-D has been studied as an alternative to the existing imperfect treatment in the ongoing Starbeam Study (ALD-102), interim data from which has been presented at the American Academy of Neurology Annual Meeting.
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