Скачать или смотреть Our Partners in this Fight: SpringWorks' FDA-Approved Drug, Gomekli (mirdametinib) for NF1

The FDA has officially approved Gomekli (mirdametinib) – the second-ever FDA-approved treatment for neurofibromatosis or schwannomatosis – and the first-ever approved for adults with NF1.

This is a game-changing moment for the NF community. For years, patients and families have waited for more treatment options. Today, that wait gets shorter.

This approval is more than just a win – it’s a result of relentless determination and collaboration. The Children’s Tumor Foundation identified mirdametinib’s potential, ensured its continued development, and partnered with SpringWorks Therapeutics to bring it to this moment. We supported recruitment for clinical trials, worked closely with SpringWorks to deepen their understanding of NF, and never stopped pushing to make this treatment a reality. And now, it is.

Read more: https://www.ctf.org/nftreatments/

The Children’s Tumor Foundation (CTF) is the drug discovery engine for NF. Everything we do is to bring treatments to patients as we work toward a cure. By connecting patients, doctors, scientists, and pharma, we drive treatments, advance care, and deliver results for millions affected by neurofibromatosis or schwannomatosis, collectively referred to as NF. Our patient-first collaborative approach accelerates drug development and brings life-changing therapies to patients faster, driven by our mission to end NF.

What is NF?
NF refers to a group of genetic conditions that cause tumors to grow on nerves throughout the body. NF affects 1 in 2,000 births and may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, or cancer. NF affects all races, ethnic groups, and genders equally. The types of NF include neurofibromatosis type 1 (NF1) and all types of schwannomatosis (SWN), including NF2-related schwannomatosis (NF2-SWN), formerly called neurofibromatosis type 2 (NF2).

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