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 ...

The MEK1 and MEK2 inhibitor showed that it was able to reduce NF1-PN lesions in the 114-patient phase 2b ReNeu trial, with an overall response rate (ORR) of 41% in adults and 52% in children.

My 10-week-old infant girl had been born with a rare condition called Neurofibromatosis Type 1 (NF1). NF1 causes tumors to grow on nerves throughout the body and can lead to blindness, learning ...

The first and only FDA-approved treatment for both adults and children 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable ...

Patients with multiple CALMs w/wo skinfold freckling and no other typical NF1 associated features (Lisch nodules, bone abnormalities, neurofibromas, optic pathway gliomas). The DNA-based ...

– Data from the pivotal Phase 2b ReNeu trial demonstrate that patients with NF1-PN achieved deep responses and improvements in health-related quality of life over the course of mirdametinib ...

NCT03962543), which evaluated the safety and efficacy of mirdametinib in patients aged 2 years and older with an inoperable NF1-associated PN causing significant morbidity (N=114). Study ...