For 2025, the company expects to continue strong commercial execution of the OGSIVEO launch in the U.S.; secure FDA approval in adults and children with NF1-associated plexiform neurofibroma.

Background: Neurofibromatosis type 1 (NF1) is a rare neurogenetic disorder with limited treatment options. Selumetinib, a MEK1/2 inhibitor, has emerged as a promising therapy for inoperable ...

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With several expansion opportunities of OGSIVEO in place, plus the ability to receive FDA approval for mirdametinib for the treatment of patients with NF1-associated plexiform neurofibromas [NF1 ...

Pathology is the precise study of the causes and consequences of a disease. The two major components of pathology are anatomical studies of structure and function at levels ranging from the whole ...

SpringWorks Therapeutics is developing a candidate for neurofibromatosis type 1-associated plexiform neurofibromas. Neurofibromatosis type 1 (NF1) is a genetic disorder that affects the growth and ...

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Amvuttra (vutrisiran) is a prescription drug that’s used to treat nerve damage related to a rare genetic condition. Amvuttra is given as an injection under your skin by a healthcare professional ...