In the Spotlight

Neurofibromatosis type 1 patient attends Camp Fantastic for the first time

Thirteen-year-old Dom was diagnosed with neurofibromatosis type 1 at birth. Because of Dom’s diagnosis, he hasn’t always had the opportunity to do the things the average teenager does, but his first year at Camp Fantastic changed that. Camp Fantastic, an annual week-long camp for pediatric cancer patients and survivors, gave Dom the opportunity to try lots of new things and some familiar ones in a setting of support, safety and friendship.

Read More

Landmark discovery: H2AX as a sensor of DNA damage

William Bonner’s curiosity about histones led to the discovery that the protein H2AX is altered in response to harmful DNA damage called a double-strand break. This powerful tool has led to breakthroughs in both basic and clinical cancer research. 

Read More

FDA grants orphan drug status to selumetinib for neurofibromatosis type 1 (NF1) treatment

The U.S. Food and Drug Administration granted orphan drug status in February to selumetinib for use in patients with the genetic disorder neurofibromatosis type 1 (NF1), who often develop tumors of the peripheral nervous system. Receiving orphan drug designation is a helpful step for selumetinib.

Read More