In NF1, many of these medications work by blocking a specific cell signaling pathway called Ras/MAPK that is hyperactive in cells that have lost NF1 function due to gene mutation. When this signaling ...

It is known that the gene product of NF1, neurofibromin, functions as a regulator of a protein called RAS. RAS is a key component of the RAS/MAPK cell signaling pathway that controls cell growth and ...

CAMBRIDGE, England--(BUSINESS WIRE)--Healx, an AI-enabled, clinical-stage biotech company dedicated to rare diseases, today announced the first patient has been dosed in INSPIRE-NF1, a Phase 2 ...