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We are pleased to present a summary of the 2018 Joint Global Neurofibromatosis Conference, held in Paris at the Maison de la Chimie on November 2 – 6, 2018.
The support and planning of this particular conference was historic in that the Children’s Tumor Foundation (CTF), for the first time, combined forces with the European Neurofibromatosis Group, Association Neurofibromatoses et Recklinghausen, and the NF2/Schwannomatosis State of the Art group, to form the most comprehensive agenda and largest gathering of NF researchers, clinicians, patients, and patient advocates, with over 800 people from around the world in attendance.
One of the more exciting developments in neurofibromatosis (NF) research today is the success of the MEK inhibitor clinical trial, in which over 70% of participants are seeing reductions of 20-55% in the size of their inoperable plexiform neurofibromas. The research surrounding this particular drug, selumetinib, is being prepared for submission to the FDA, and indicators are that it could become the first-ever approved drug for NF.
Your ongoing support has allowed us to take huge steps forward in 2018 in the fight against NF. Your efforts and contributions have been instrumental in advancing neurofibromatosis research, providing hope for the millions worldwide who live with NF, and bringing us ever closer to our shared goal of ending NF forever.
Major Research Funders Join Together to Advance Research into Genetic Disorder
New York/Baltimore/Seattle – The Children’s Tumor Foundation (CTF), together with the Neurofibromatosis Therapeutic Acceleration Program (NTAP) and Sage Bionetworks (Sage), has announced the first-ever open data portal for scientific research results in the field of neurofibromatosis (NF). The NF Data Portal marks the major first milestone in all three organizations’ commitment to the development of the larger NF Open Science Initiative (NF-OSI), which draws experts from across research disciplines and disease areas in order to develop treatments for NF, a genetic disorder which affects 2.5 million people worldwide (1 in 3,000 births), and currently has no cure or effective treatment options.
Congratulations to our friends at the Gilbert Family Foundation on the announcement of the launch of their Gene Therapy Initiative in NF1. With a substantial infusion of $12 million in research funding to top researchers over the next three years, the grants will support research that address the underlying genetic causes of NF1. This includes gene replacement, gene editing, RNA editing, exon skipping, and nonsense mutation suppression.