Neurofibromatosis type 1 (NF1) is a rare genetic condition that affects how certain nerve cells grow and develop. Children with NF1 can have small growths on or under the skin called neurofibromas.
This month, our discussion focuses on the clinical management of plexiform neurofibromas in the era of selumetinib, a MEK inhibitor medication that was approved by the FDA in 2020 for the treatment of ...
Almost half of adults and children with neurofibromatosis type 1 (NF1) responded to the investigational MEK inhibitor mirdametinib, a large multicenter study of the rare condition showed. Overall, 53 ...
Targeted therapies called MEK inhibitors are changing the treatment landscape for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). NF1-PN are benign tumors that develop in the ...
Figure 1 (A) Clinical picture, (B) Usg showing intramuscular heterogenous lesion arising from sciatic nerve, (C) MRI showing heterogeneously isointense mass measuring 12.8 × 6.6 × 3.8 cm with features ...
I’d like to discuss here the evolving therapeutic landscape in NF1. For the past several years, the NF scientific community, including the UAB NF Research Program, has put major emphasis on conducting ...
UCLA researchers developed a new method to study NF1, accelerating the search for potential treatments. Future work is supported by $1.6 million in grants from the Neurofibromatosis Therapeutics ...
Q: A doctor said the rashes on my son's back were “coffee spots” and that meant he might have neurofibromatosis. I read there is a new treatment for this condition; is it a cure? A: Café-au-lait spots ...
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 trial ...
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