BioMarin Pharmaceutical’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study.
BioMarin’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study.
Patients with the condition don’t produce …
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Tested in a condition known as ENPP1 deficiency, the treatment failed a trial goal that’s important to regulators, spurring…
Though two serious side effects muddied the results, CEO Curran Simpson expressed optimism about a clearance and claimed FDA…
Regenxbio said Thursday that its Duchenne muscular dystrophy gene therapy met the bar in a pivotal study, and it’s aiming for FDA…
A closely watched therapy developed by Johnson & Johnson failed to show a statistically meaningful improvement for IBD patients.
BioMarin Pharmaceutical said on Friday it would acquire Amicus Therapeutics for about $4.8 billion, expanding its presence in…
Early last year, startup iECURE’s experimental gene therapy seemingly cured a baby of a life-threatening genetic disease. But new…
