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FDA Approves Adzynma for Congenital Thrombotic Thrombocytopenic Purpura
The FDA announced it has approved Adzynma as prophylactic or on-demand enzyme replacement therapy for the treatment of congenital thrombotic thrombocytopenic purpura.
Peter marks
Speror cataland
Wexner medical center
Congress spotlight
Ohio state university
Us thrombotic microangiopathy alliance
Ohio state university speror cataland
Drug administration
Takeda pharmaceuticals
Rare pediatric disease priority review voucher
Priority review
Fast track
Biologics evaluation
First treatment
Rare inherited blood clotting
Pivotal phase
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