neurofibromatosis

Breakthrough Treatment Brings Hope to Children with Tumor Disorder

Brigitte Widemann Recognized for Pioneering Work on Debilitating Disease

Dr. Brigitte C. Widemann

Getting diagnosed with a serious illness as an adult can be devastating, so one can hardly imagine the impact of receiving such news as a child, particularly when the disease has no good treatments. Until recently, this was the case for many children with the potentially severe and frequently disfiguring condition neurofibromatosis type 1 (NF1). However, pioneering research led by IRP senior investigator Brigitte C. Widemann, M.D., led to the first-ever drug approved by the U.S. Food and Drug Administration (FDA) to treat the condition. For this groundbreaking work, Dr. Widemann, her IRP research team, and her collaborators outside NIH were named as finalists for the 2020 Samuel J. Heyman Service to America Medals, also known as the ‘Sammies,’ an award that honors exceptional work by government employees.

Need for Speed

Reblog via The Children's Inn at NIH: Family Stories.

Travis Carpenter NF1

From Travis’ appearance and attitude, you’d never believe that, inside his body, many things are wrong. His legs are different lengths, his bones are prone to breaking, and he has a long, “deep” tumor running from his lower spine down across his hip to below his knee. He also has lower back pain from constant irritation to the nerves in his spine.

Meet Children's Inn Resident Connor O’Brien

Reblog via The Children's Inn at NIH: Family Stories.

Connor, Childrens Inn at NIH

In the words of Connor: “A lot of times treatment for cancer and chronic diseases is very difficult to sustain. A lot of times it hurts. A lot of times you have to be given anesthesia, invasive things like that. The Inn gives you somewhere to come home to, somewhere to end your day, a place where you can have closure. Thank you all for making sure we have The Inn to come home to.”