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G. Castaman and D. Matino et al.
improving the clinical outcome and, even more so, the quality of life of HB patients when compared to HA patients. The promising perspectives of gene therapy are painting a future scenario in which the decision to offer this option to patients with HB and very mild clinical problems could be challenging considering the costs involved and the yet unknown long-term effects.
Acknowledgments
GC has received unrestricted research grants directly to his Institution by CSL Behring, Pfizer and Sobi and has received honoraria as a speaker or for participating in Advisory Boards or educational events from Bayer, Baxalta/Shire, CSL Behring, Kedrion, Novo Nordisk, Pfizer, Roche, Uniqure.
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