Living with a Rare Disease: A Pastor’s Journey Finding the Right Care and Treatment for NF1-PN
STAMFORD, Conn., March 02, 2026 (GLOBE NEWSWIRE) -- Did you know neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) is a rare genetic disorder that can cause tumors to grow along nerves, leading to pain, mobility challenges, and lifelong complications? 1 This rare condition affects approximately 30,000 to 50,000 people in the United States, including Austin, who has lived with NF1-PN since early childhood. 1-5
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Living with NF1-PN has affected nearly every aspect of Austin’s life, from repeated surgeries at a young age, to pain and other symptoms which forced him to give up his career in law enforcement. As he grew older, Austin learned to advocate for himself, working closely with his doctors to manage symptoms and explore treatment options. After consulting with his care team, Austin decided to participate in a clinical trial for what is now FDA-approved as GOMEKLI ® (mirdametinib) for treating symptomatic, inoperable PNs in adults and children with NF1. Please see important safety information and Patient Information for GOMEKLI.
Austin shares his experience to help others feel less alone and encourage those newly diagnosed to speak up, seek specialized care, and connect with the NF1-PN community for more information and resources.
Learn more: https://www.gomekli.com/
What is GOMEKLI?
GOMEKLI (mirdametinib) is a prescription medicine used to treat adults and children 2 years of age and older with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas that cause symptoms and cannot be completely removed by surgery.
It is not known if GOMEKLI is safe and effective in children under 2 years of age.
IMPORTANT SAFETY INFORMATION
Before taking GOMEKLI tell your healthcare provider about all of your medical conditions, including if you:
Males with Female partners who are able to become pregnant:
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
How should I take GOMEKLI?
See the “Instructions for Use” that come with your medicine for instructions on how to prepare and take GOMEKLI tablets for oral suspension.
What are the possible side effects of GOMEKLI?
GOMEKLI may cause serious side effects, including:
The most common side effects of GOMEKLI in adults include:
The most common severe abnormal blood tests in adults include an increased enzyme called creatine phosphokinase (CPK).
The most common side effects of GOMEKLI in children include:
The most common severe abnormal blood tests in children include decreased white blood cell (neutrophil) counts and increased CPK.
GOMEKLI may cause fertility problems in females, which may affect your ability to have children. Talk to your healthcare provider if you have concerns about fertility.
These are not all of the possible side effects of GOMEKLI. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Please click here for full Prescribing Information including Patient Information and Instructions for Use.
References:
C_GOM_US_0564 02/26
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