BioMarin will discontinue dosing and enrollment of its phase 2 trials for Voxzogo—currently approved in achondroplasia, a ...
BioMarin said the hip injury cases haven't appeared in its own phase 2 studies, nor among the more than 5,000 children treated with the drug for achondroplasia--a common form of dwarfism--over the ...
March 16 (Reuters) - BioMarin Pharmaceutical will discontinue mid-stage trials of its bone disorder treatment after reports of several cases of patients' hip joint slipping out of place, the drugmaker ...
Management prioritizes 5%–10% weight loss and acetazolamide titration guided by Frisén grading, OCT, and visual fields; refractory or vision-threatening disease may need ONSF or CSF shunting.
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