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 ...

Neurofibromatosis type 1 (NF1) is the most common of the three types of neurofibromatosis ... Most people with NF1 develop symptoms before age 10, though some symptoms may not appear until puberty.

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 ...

My 10-week-old infant girl had been born with a rare condition called Neurofibromatosis Type 1 (NF1). NF1 causes tumors ... I teamed up with the Children’s Tumor Foundation, an organization ...

In April 2024, Healx signed an investment agreement with its long-term partner Children's Tumor Foundation ... first subject ...

SpringWorks Therapeutics and the Children’s Tumor Foundation salvaged a shelved asset for neurofibromatosis and got it over ...

The University of Pittsburgh receives $1 million from The Giorgio Foundation to establish an endowed chair for ...

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 ...

has become the first medicine approved for both adults and children with plexiform neurofibromas (PN) in patients with the genetic disorder neurofibromatosis type 1 (NF1), a rare genetic condition.

These neurofibromas are soft to the touch, painless, and usually appear as small, pink or purple bumps on the skin. Most young children rarely have more than a few small skin neurofibromas. Additional ...