Indication

Neurofibromatosis type 1 with plexiform neurofibromas

Neurofibromatosis type 1 with plexiform neurofibromas refers to an autosomal dominant genetic tumor predisposition syndrome characterized by the development of complex, benign peripheral nerve sheath tumors. Read more about Neurofibromatosis type 1 with plexiform neurofibromas

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Common Neurofibromatosis type 1 with plexiform neurofibromas therapies.

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KOSELUGO (selumetinib sulfate)

ANTINEOPLASTIC - MEK1 AND MEK2 KINASE INHIBITORS·NEUROFIBROMATOSIS TYPE 1 WITH PLEXIFORM NEUROFIBROMAS
AstraZeneca Pharmaceuticals

KOSELUGO (selumetinib sulfate)

ANTINEOPLASTIC - MEK1 AND MEK2 KINASE INHIBITORS·NEUROFIBROMATOSIS TYPE 1 WITH PLEXIFORM NEUROFIBROMAS
AstraZeneca Pharmaceuticals

GOMEKLI (mirdametinib)

ANTINEOPLASTIC - MEK1 AND MEK2 KINASE INHIBITORS·NEUROFIBROMATOSIS TYPE 1 WITH PLEXIFORM NEUROFIBROMAS
SPRINGWORKS THE
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About Neurofibromatosis type 1 with plexiform neurofibromas.

Neurofibromatosis type 1 with plexiform neurofibromas refers to an autosomal dominant genetic tumor predisposition syndrome characterized by the development of complex, benign peripheral nerve sheath tumors.

Driven by pathogenic variants in the *NF1* gene that lead to loss of the tumor suppressor neurofibromin and hyperactivation of the RAS-MAPK signaling pathway, these tumors arise primarily from Schwann cells and expand along single or multiple nerve fascicles and branches.

Plexiform neurofibromas typically manifest in early childhood, affecting deep or superficial nerves across various anatomic sites.

Because they infiltrate surrounding tissues, they frequently cause severe pain, motor deficits, spinal or skeletal deformities, and airway or vascular compromise, while also harboring a lifelong risk of malignant transformation into peripheral nerve sheath tumors.

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