Experimental Gene Therapy for Hemophilia B: Bill’s Story
Bill’s hemophilia caused several serious health crises. After nearly 60 years of continual treatments, a gene therapy transformed his life.
Patient stories
Hemophilia Patient Stories
Experimental Gene Therapy for Hemophilia B: Jay’s Story
For 50-plus years, Jay dealt with the frequent treatments needed for his hemophilia. A gene therapy put an end to the treatments and to the worry the disease caused him.
Experimental Gene Therapy for Hemophilia B: Bill’s Story
For nearly 50 years, Bill needed frequent treatment for hemophilia — often every few days. A gene therapy changed everything.
Goldenhar Syndrome & Von Willebrand Syndrome: Aidan's Story
Aidan was born with Goldenhar syndrome and Von Willebrand syndrome, two rare genetic conditions that need expert medical management. He receives care from The Children's Hospital of Philadelphia at CHOP Specialty Care Centers in New Jersey.