BioMarin Reports ‘Promising’ Results In Treatment For Deadly Genetic Disease
The pharmaceutical company said its clinical trial slowed the rate of decline for some patients with Batten disease, which starts with a slowing of speech and seizures and progresses to blindness and immobility. Most children with it die by the time they are 12, and there are currently no treatments for the disease.
The San Francisco Business Times:
'Kids On The Cusp': Early Data Moves BioMarin Against A Childkiller
Children with a genetic mutation that robs them of their speech, muscles and lives could get access to an experimental treatment from BioMarin Pharmaceutical Inc. yet this year. The San Rafael-based rare disease drug developer said Wednesday that an early-stage clinical trial indicated that its enzyme replacement therapy — called cerliponase alfa, or BMN-190 — slowed the rate of decline in most patients with CLN2 disease, a form of deadly Batten disease. (Leuty, 3/3)