
What Is an Orphan Drug? A Guide for Families Facing a Rare Disease
Key Takeaways: An orphan drug treats a rare disease. In the United States, that means a condition affecting fewer than 200,000 people. Most rare diseases

Key Takeaways: An orphan drug treats a rare disease. In the United States, that means a condition affecting fewer than 200,000 people. Most rare diseases

Key Takeaways: Neurofibromatosis is a group of genetic conditions. They cause tumors to grow along nerves. Most of these tumors are benign, but they need

Key Takeaways: The plan is named for how care is delivered. The C4K Way, Every Day is built on the idea that the experience of

Key Takeaways: Pediatric oncology nurses are often the most consistent presence on a child’s cancer care team. They administer chemotherapy and other treatments, monitor for

Key Takeaways: Chronic joint pain, swelling, or stiffness in children may need evaluation by a pediatric rheumatologist. This type of specialist is trained to diagnose

Key Takeaways: Pediatric hematology is the medical specialty focused on blood disorders in children. These conditions affect red blood cells, white blood cells, platelets, and

Key Takeaways: Your child’s cancer care involves a full team, not a single doctor. A pediatric oncology care team typically includes oncologists, nurses, nurse practitioners,

Key Takeaways: Pediatric cancer clinical trials are carefully designed, federally regulated research studies that test new or improved treatments for children with cancer. Child cancer

Key Takeaways: Gene therapy for hemophilia B targets the root cause of the condition — the faulty gene — rather than replacing the missing clotting