1947
Dr. Alfredo Pavlovsky, a doctor in Buenos Aires, Argentina, distinguishes two types of hemophilia in his lab – A and B.
Image courtesy of CSL Behring
1952
Image courtesy of CSL Behring
Stephen Christmas is diagnosed with hemophilia B, which doctors colloquially refer to as Christmas Disease. Fresh frozen plasma is the only treatment option available, and the average life expectancy is less than 20 years old.
Image courtesy of CSL Behring
Stephen Christmas is diagnosed with hemophilia B, which doctors colloquially refer to as Christmas Disease. Fresh frozen plasma is the only treatment option available, and the average life expectancy is less than 20 years old.
Patient
Dan has experienced the full treatment spectrum for hemophilia, from lyophilized plasma to factor. Now with a young grandson who also has hemophilia, he’s amazed by how far we’ve come and is excited for where the science is headed with gene therapy.
Dan's story
Image courtesy of Gary Bottoms
1970s
Image courtesy of Gary Bottoms
Factor concentrates in powdered form become available, allowing for home infusions and significant reduction in treatment time.
Researchers pioneer genetic engineering, creating the potential to treat genetic conditions by modifying a person’s genes.
Image courtesy of Gary Bottoms
Factor concentrates in powdered form become available, allowing for home infusions and significant reduction in treatment time.
Researchers pioneer genetic engineering, creating the potential to treat genetic conditions by modifying a person’s genes.
Patient
Christopher
Christopher was told he wouldn’t live past age 5 — but after years of health crises, he is hopeful for his future and that of his children due to the advancement of hemophilia treatments.
Christopher's Story
Healthcare Provider
Sue
Sue, a now-retired nurse coordinator, spent the last three decades on the front lines caring for people with hemophilia.
Sue's story
Image courtesy of Sue Geraghty
1980s
Image courtesy of Sue Geraghty
Scientists successfully clone the Factor VIII and IX genes, paving the way for breakthrough synthetic factor therapies (not derived from human plasma) using recombinant technologies.
It is discovered that much of the human-derived clotting factor had come from blood donations contaminated with blood-borne diseases, such as HIV and hepatitis viruses.
Image courtesy of Sue Geraghty
Scientists successfully clone the Factor VIII and IX genes, paving the way for breakthrough synthetic factor therapies (not derived from human plasma) using recombinant technologies.
It is discovered that much of the human-derived clotting factor had come from blood donations contaminated with blood-borne diseases, such as HIV and hepatitis viruses.
Scientist
Ying Poi
As a scientist working in gene therapy, Ying Poi has seen the many advancements of hemophilia treatments firsthand.
Ying Poi's story
Image courtesy of Thomas Wilkes
1990s
Image courtesy of Thomas Wilkes
The first recombinant Factor IX products are approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) to treat hemophilia B, leading the way for prophylactic treatment, which dramatically improve patients’ lives.
Hemophilia patient life expectancy improves to 67 years old.
The beginning rounds of in-human hemophilia gene therapy trials are initiated utilizing a variety of methods.
Image courtesy of Thomas Wilkes
The first recombinant Factor IX products are approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) to treat hemophilia B, leading the way for prophylactic treatment, which dramatically improve patients’ lives.
Hemophilia patient life expectancy improves to 67 years old.
The beginning rounds of in-human hemophilia gene therapy trials are initiated utilizing a variety of methods.
Patient
Adam
A child of the 90s, Adam felt comfortable engaging in most activities with family and friends thanks to the availability of prophylactic hemophilia treatments.
Adam's Story
Healthcare Provider
Dr. Klamroth
A leader in the hemophilia medical community, Dr. Klamroth has witnessed several major treatment advancements over the past 25 years and looks forward to what the next 25 years will bring.
Dr. Klamroth’s Story
2000s
Evidence grows for the potential of adeno-associated virus (AAV) vectors to deliver safe, effective, and lasting genes that produce blood-clotting factor into the body.
Evidence grows for the potential of adeno-associated virus (AAV) vectors to deliver safe, effective, and lasting genes that produce blood-clotting factor into the body.
Patient
Jeron
Though held back from playing sports as a child, Jeron has channeled his energy into being an advocate in the hemophilia community, traveling the world and inspiring others with the condition.
Jeron's Story
Image courtesy of Jennifer Mermilliod
2010s
Image courtesy of Jennifer Mermilliod
Long-acting recombinant factor therapies are available offering the hemophilia community additional treatment options.
Late-stage trials for AAV-based gene therapy in hemophilia begin.
Image courtesy of Jennifer Mermilliod
Long-acting recombinant factor therapies are available offering the hemophilia community additional treatment options.
Late-stage trials for AAV-based gene therapy in hemophilia begin.
Patient
Nathan
Nathan credits his happy and full childhood to his mother and his prophy regimen. Now a college student, Nathan’s journey with hemophilia has inspired him to pursue a medical degree.
Nathan’s Story
Caregiver
Jennifer
Having a child with hemophilia added another layer of worry to being a mother, but Jennifer raised her son with resilience, strength, and a dash of gratitude.
Jennifer’s Story
Patient/Caregiver
Stormy
As a woman with hemophilia and a caregiver to a son with hemophilia, Stormy has made it her mission to educate others, advocating for men and women alike in the hemophilia community.
Stormy's story
Image courtesy of Mike Hargett
2020s
Image courtesy of Mike Hargett
Gene therapy trials in hemophilia continue and show promise in potentially decreasing annualized bleeding rates and use of replacement therapy.
The U.S. Food and Drug Administration (FDA) approves gene therapies for the treatment of hemophilia B (2022 and 2024) and hemophilia A (2023).
Image courtesy of Mike Hargett
Gene therapy trials in hemophilia continue and show promise in potentially decreasing annualized bleeding rates and use of replacement therapy.
The U.S. Food and Drug Administration (FDA) approves gene therapies for the treatment of hemophilia B (2022 and 2024) and hemophilia A (2023).
Patient
Wayne
As an advocate for hemophilia for over 40 years, Wayne has experienced the monumental advancements in hemophilia treatments and has committed his life to ensuring a better world for today’s hemophilia community.
Wayne's story