The critical need for postmarketing surveillance in gene therapy for haemophilia.

Barbara A KonkleMichael RechtAnneliese HilgerPeter Marks

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2020)

Support of all stakeholders in gene therapy, providers, patients, industry and regulators, augers successful capture of uniform long-term safety and efficacy data to ensure optimal treatment of people with haemophilia.

Keyphrases

gene therapy
end stage renal disease
ejection fraction
newly diagnosed
public health
peritoneal dialysis
prognostic factors
electronic health record
gene expression
patient reported outcomes
dna methylation
replacement therapy
genome wide analysis