On May 1, 2017, an international consortium of clinicians and scientists from multiple disciplines met in Toronto, Canada to officially launch the Synodos for Schwannomatosis project. Led by Dr. Gelareh Zadeh from University of Toronto and Dr. Laura Papi from University of Florence, Italy, the project aims to perform an extensive molecular analysis of schwannomatosis tumor samples to identify new therapeutic targets, and advance the understanding and management of the disease, with special focus on pain.
Mark your calendar: the 2018 NF Conference will be taking place November 2-6, 2018 at the Maison de la Chimie in Paris, France. As details become available, they will be posted at www.ctf.org/nfconference.
Neurofibromatosis Therapeutic Acceleration Program (NTAP) is launching a new research program focused on understanding the pathogenesis of cutaneous neurofibromas. NTAP is a philanthropic research entity based at Johns Hopkins University dedicated to the discovery of therapeutics for the peripheral nerve tumors that afflict people with neurofibromatosis type 1 (NF1). Cutaneous neurofibromas represent a major unmet need for NF1 patients. This research program sets the blueprint for improving therapeutics for these tumors by encouraging research proposals that improve the Understanding of Cutaneous Neurofibromas.
The Family Center for Neurofibromatosis at Massachusetts General Hospital is conducting a research study aimed at helping adults with NF2 who are deaf or have severe hearing loss manage stress associated with NF2
Every year, on the last day of February, we recognize Rare Diease Day, a time for the entire rare disease community to come together to raise awareness. NF affects 1 in 3,000 people overall; NF2 is even more rare (1 in 25,000) and schwannomatosis even more rare (1 in 40,000). These days, there is a lot of excitement and hope for the MEK clinical trial for inoperable NF1 plexiform tumors (click here to read about the trial). Over 70% of patients had a dramatic response and saw their tumors shrink 20% or more. This is unprecedented and really makes us hope that this drug will soon be approved by the FDA as the first-ever treatment for NF1. But what about NF2? Even though it's the MEK trial making headlines in NF these days, there is a lot going on also for NF2. In recognition of Rare Disease Day, we're sharing these stories about two adults living with NF2 and their experiences in the Avastin (Bevacizumab) trial.