CSL Behring has closed an agreement giving it global commercialization and licensing rights to AMT-061, an experimental gene therapy for hemophilia B now being tested in a Phase 3 clinical trial. The therapy’s developer, uniQure, will receive an upfront cash payment of $450 million from CSL…

Sobi and Sanofi’s philanthropic organization, Foundation S, have announced an additional donation of up to 100 million international units (IUs) of clotting factor treatment to support the World Federation of Hemophilia (WFH) Humanitarian Aid Program. This is the second renewal of the 10-year partnership that will continue…

A new mom recently called me to share her concerns and to ask questions about choosing a new treatment for her son. She needed a listening ear, and just thinking about the possibility that her little boy might never need an implanted port gave me chills. I thought…

Katherine High, MD, has received the gold medal from Children’s Hospital of Philadelphia (CHOP) in recognition of her work to advance gene therapies for hemophilia and other genetic disorders. In CHOP’s 166-year history, this award — to recognize the most significant achievements in improving the health of children —…

Tuesday morning, March 8. Bright rays of sun had already passed through our floor-length bedroom windows, but my sleep-deprived brain was fighting to keep my eyes shut for a few more minutes. The day before I had worked myself into a mental breakdown, which had zapped my energy completely. “Wake…

The Institute for Gene Therapies (IGT) has launched, with the aim of maximizing the potential of gene therapies in genetic disorders such as hemophilia. Comprised of industry leaders, scientists, and patient advocates, the IGT’s overarching goal is to set the foundation for a modernized regulatory and reimbursement…

As part of a Coalition for Hemophilia B contest, people with hemophilia B, their siblings, and caregivers are  invited to design a comic or coloring book that tells a story about how they manage this rare blood disease. There will be a $500 award for winners in each of…

My wife and I sat by the pool and watched our 3-year-old super fish glide through the water. The skill he displayed with a set of floaties wrapped around his arms took our breath away. Our son became one with the water as he swam across the bright blue surface.

The World Federation of Hemophilia (WFH) is commemorating having successfully distributed, since 1996, over one billion international units (IUs) of factor and non-factor replacement therapies for people with inherited bleeding disorders, such as hemophilia, through its Humanitarian Aid Program. Donations from the program have improved the quality…

Sobi and Sanofi announced an additional donation of up to 500 million international units (IUs) of clotting factor therapy in support of the World Federation of Hemophilia‘s Humanitarian Aid Program. The clotting factor goes toward treating people with hemophilia in developing countries where access to medicine is…