Paroxysmal Nocturnal Hemoglobinuria (PNH)
Science Simplified: What is a Natural History Study?
Surfer grounded by PNH Treatment but Thriving!
I was diagnosed with PNH fifteen years ago, at the age of 51. This is my story.
I grew up on the West Side of Los Angeles. Raised by a single parent, my mother was a strong woman of faith. My father, the eldest of twelve, died of a heart attack when I was about nine months old. Four of his six brothers, and a couple of my cousins died at an early age. My mother always said, “those Harness men have bad blood.” Little did I know what she meant at the time.
Fighting for a Transplant : Stefania conquers Aplastic Anemia and PNH ~ Stefania demuestra su VERDADERA fuerza
Hi! My name is Stefania, I am 31 years old, and I am from Ecuador.
Emma Groarke, MD, FRCPath
Genetic Testing for Bone Marrow Failure Diseases
Pediatric PNH - Diagnosis and Treatment
Satheesh Chonat, MD
Clinical Trials and Children
Until recently, children were rarely included in studies of medical treatments. As a result, much is still unknown about how children respond to drugs, some biologics (such as gene therapy) and medical devices. According to the U.S. Food and Drug Administration (FDA), only 20-30% of approved drugs have actually been labeled for use in children.