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Males With Hemophilia A Report Physical, Mental Burdens of Disease Regardless of Severity

Self-reported data from adult and pediatric males with hemophilia A show that burden of the disease persists regardless of severity, highlighting a need for improved prophylactic treatment. ....

Image Credit , Hemophilia A , Prophylactic Treatment , Target Joints , Chess Paeds ,

Buzz for gene therapies is loud, but drugmakers struggle to get treatments off the ground

The current state of Roctavia, the first gene therapy for treating hemophilia A, and the struggles faced by drugmakers in the gene therapy market ....

Biomarin Pharmaceutical , Hemophilia A , Gene Therapy , Biomarin Pharmaceutical , Inherited Disorders , 00 Million ,