On April 10, 2020, the Food and Drug Administration approved selumetinib (KOSELUGO, AstraZeneca) for pediatric patients, 2 years of age and older, with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). Selumetinib, a kinase inhibitor, is the first therapy approved for pediatric patients who have this...
Research
Research related to Neurofibromatosis.
NF Registry
If you haven’t signed up for the NF Registry you can use this link to get registered. This patient-centered database allows those living with any form of NF to contribute to research and stay up-to-date on studies of potential treatments.
Pfizer Spin Off To Study NF
Pfizer spins out SpringWorks Therapeutics to focus on diseases like Neurofibromatosis. Read more at “Pfizer Creates New Smaller Company to Focus on Rare Diseases“.
Clinical Trial of Selumetinib
A phase I clinical trial of Selumetinib has shown some early promising results in children diagnosed with NF1. read more at “New Drug Shrinks Pediatric Tumors“.
New Study for Coping with Pain
Coping with Pain: an ACTive Approach Do you have NF1 and one or more plexiform neurofibromas? Do you have pain that has lasted three months or more? Are you between the ages of 16 and 34 years? If you answered yes to all these questions, you may be eligible...
Senate Bill Threatens NF Research
Senate bill threatens Neurofibromatosis research again! Use this link to learn more and take action now. Thanks.
NF Funding At Risk of Elimination
Senate amendment threatens Neurofibromatosis research. Take action today! The Senate is currently considering the National Defense Authorization Act for Fiscal Year 2016 (HR 1735). Sen. John McCain (R-AZ) has filed an amendment that, if passed, would severely restrict the types of medical research that could be funded by the...
Federal Funding for NF Research
You can contact Congress by March 20 to let them know that federal funding for NF research is important to you. See the message below for a little more info and use this link to “Voice Your Support For NF Research“. Dear NF Friends Old and New, Every March...
Sign Up For NF Registry
If you or your child have NF1, NF2, or Schwannomatosis, please consider signing up for the NF Registry using this link. It was created by the Children’s Tumor Foundation in 2012 to serve as a resource for information on NF. Each registration can help them build and strengthen the NF...
Welcome To Our Blog
Welcome to the blog for the Neurofibromatosis Clinics Association (NFCA) of Pittsburgh, a regional, non-profit dedicated to serving people with NF and their families. Stay tuned for posts news and regional events related to Neurofibromatosis. If you’re a patient, family member, donor, volunteer or healthcare professional give us a follow.