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 ...

Gomekli (mirdametinib) is the first treatment that is FDA approved for both adults and children with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas (PN). Selumetinib, a drug that ...

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.

The drug is being evaluated in adult NF1 patients who have symptomatic, inoperable plexiform neurofibromas, or PN, according to a statement. AstraZeneca (AZN) and Merck (MRK) are jointly ...

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 ...

It’s important to know that an accurate diagnosis of NF1 can only be made by a physician with expertise in the diagnosis and treatment of neurofibromatosis. Because people with NF1 often have multiple ...

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 ...

– 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 ...