Panelists discuss how NF1-associated plexiform neurofibromas can cause significant morbidity, including disfigurement, pain, and neurologic impairment, with the risk of malignant transformation, and ...

Michelle and Samantha Pearson are a mother-daughter duo diagnosed with NF1-PN, a rare and painful disease that causes benign ...

Panelists discuss how neurofibromatosis type 1 (NF1) is typically diagnosed in early childhood using National Institutes of ...

The neurofibromatosis type 1 market remains niche, but awareness campaigns, increased diagnoses, and treatment adoption, along with ongoing clinical ...

Adults with NF1-PN have no approved therapies. Additionally, selumetinib is available only in tablets, which limits use in young children and patients with swallowing difficulties. Mirdametinib is ...

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 company was already making progress to further expand the use of this MEK inhibitor into another territory for the treatment of adults and children with NF1-PN. As a matter of fact ...

Its only competitor at the moment is AstraZeneca's MEK 1/2 inhibitor Koselgo/Koselugo (selumetinib), co-marketed by MSD, which was approved for NF1-PN in paediatric patients aged two and older in ...

– 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 ...