Our Mission To provide information, education, advocacy, and direct assistance to and on behalf of people with bleeding disorders, and to encourage and support scientific research to improve medical treatments and develop cures for hemophilia and related disorders. Learn More ...
These new therapies offer many options for improved treatment and even cures of these disorders, but will require ongoing study to determine their optimal use and ensure safety. Authorship Contribution: M.U.C., R.S., and S.W.P. contributed equally to the concept, writing, and editing of ...
and a deep understanding of the significant challenges that people living with hemophilia continually face. We are proud to advance the latest innovation for people living with hemophilia B and are encouraged by the potential of this investigational gene therapy," ...
Pfizer and Spark Therapeutics entered into a License Agreement in December 2014 for the hemophilia B gene therapy programme. Under the terms of the agreement, Pfizer will now assume sole responsibility for all subsequent pivotal studies, all regulatory activities, manufacturing and global commercialization...