The FDA approved the enzyme replacement therapy Adzynma for the prophylactic or on-demand treatment of adults and pediatric patients with congenital thrombotic thrombocytopenic purpura, a rare blood ...

(51 of 54) remained free from the burden of continuous prophylaxis treatment through five years following a single infusion of HEMGENIX, demonstrating sustained therapeutic benefit- At year five, mean ...

Adzynma is the first product to be approved for the treatment of the rare blood disorder that has 90% mortality when left untreated. Congenital thrombotic thrombocytopenic purpura (cTTP) can now be ...

FDA reports rare neutralizing antibodies, including a pediatric death, in patients treated with Takeda's Adzynma for congenital TTP.

Adzynma is a purified recombinant form of the ADAMTS13 protein designed to replace the missing or deficient ADAMTS13 enzyme. The Food and Drug Administration (FDA) has approved Adzynma for ...

Takeda Pharmaceuticals' drug Adzynma is being investigated by the U.S. Food and Drug Administration due to a death following treatment. The FDA said Friday it has received reports of neutralizing ...

Prophylactic cardiac treatment in DMD patients is associated with a 54% lower hazard of death, highlighting its potential survival benefit. Only 27.7% of DMD patients received prophylactic cardiac ...

Heparin is widely used as an anticoagulant, but evokes in some patients a potentially life-threatening condition called HIT. Clinical scientists at Ludwig-Maximilians-Universitaet (LMU) in Munich have ...

Sometimes in experimental biology the very factor that is part of the disease-causing mechanism can also be the factor that helps to counterbalance the disease itself. This is the case with a new ...