Different types of assays led to different results in measures of factor IX variant FIX-R338L, the authors found. A new field study could help clinicians better interpret laboratory measurements of ...
The gene therapy improved annualized bleed rates, though a handful of study participants resumed factor IX prophylaxis. A new report says the gene therapy fidanacogene elaparvovec (Beqvez) appears to ...
FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to normalize factor IX levels in patients ...
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