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BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that an individual in Germany with severe hemophilia A was treated with ROCTAVIAN® (valoctocogene roxaparvovec-rvox), marking the first time that the gene therapy has been...
All registration end points remained well above injection baseline at year 4, except for VA at year 4 in the OI group, because 1 patient experienced a retinal detachment. End point improvements included stable gains in ambulatory navigation and light sensitivity, the end points most representative ...
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