You can make a difference in improving care for people with NF1. If you or your child has NF1 and haven’t visited an NF Clinic Network Clinic (NFCN) in the last twelve months, you may be eligible to participate in this important research study. The goal is to enhance the care that people with NF1…
Read MoreFDA Grants Priority Review for Mirdametinib for the Treatment of Adults and Children with NF1-PN
Posted By NF Midwest | On August 28th, 2024 Press Release FDA Grants Priority Review to SpringWorks Therapeutics’ New Drug Applicationfor Mirdametinib for the Treatment of Adults and Children with NF1-PN– PDUFA target action date of February 28, 2025 –– EU Marketing Authorization Application also validated by European Medicines Agency – STAMFORD, Conn., August 28,…
Read MoreExisting Drug Shows Promise as Treatment for NF2-Related Schwannomatosis
There’s some exciting news for those with NF2-related schwannomatosis (NF2-SWN) that might bring hope to treatment options! A recent study explored the use of Brigatinib, a medication already used for lung cancer, as a potential treatment for NF2-SWN. The research, led by Dr. Scott Plotkin and a team of experts from various institutions like NYU…
Read MoreNF1 Optic Pathway Gliomas Study
Identify biomarkers for visual restoration strategies in Neurofibromatosis Type 1 optic pathway glioma patients. Stanford Ophthalmology, Children’s Hospital of Philadelphia and Gilbert Family Foundation | 2024 This study is seeking to learn how different eye tests and devices can help understand vision in patients with NF1, including how vision loss might show up on different…
Read MoreNF Midwest Co-Funds New NF2 Gene Therapy Project
Thanks to the support of our donors, NF Midwest is proud to be one of four NF organizations collaborating to fund a new NF2 Gene Therapy project. NF Midwest, NF2 BioSolutions, NF Northeast, and NF Michigan have combined forces to fund a USD$100,000 Gene therapy project at Dr. Wade Clapp’s Lab at the Indiana University….
Read MoreNF Midwest Collaborates to Fund NF2 Gene Therapy Research
NF Midwest, NF Northeast and NF2 BioSolutions have joined forces to fund an NF2 Gene Therapy Research Study at the Mueller Lab at the Gene Therapy Center, UMass Medical School. This $65,000 grant will be used to design, manufacture and test potential gene therapy vectors for Neurofibromatosis Type 2. Dr. Katharina Meijboom, a postdoctoral researcher, is heading up the project. Her…
Read MoreArticle: Study Shows More Fatigue in Neurofibromatosis Type 1
An interesting article was just published in the Journal of Paediatric and Child Health called “Perceived fatigue in children and young adults with neurofibromatosis type 1”. This was a small study done in the United Kingdom that compared 75 children with neurofibromatosis type 1 to their 16 unaffected siblings to determine if fatigue was more…
Read MoreNew Study Results on Pain and Patients with Neurofibromatosis Type 1
Frank Buono PhD, from the Department of Psychiatry at Yale School of Medicine and longtime member and supporter of NF Midwest, just published an article on pain and neurofibromatotis type 1 in the Journal of Pain Research called Pain symptomology, functional impact, and treatment of people with Neurofibromatosis type 1. Thank you to those in…
Read MoreNF Midwest Work Aids Development of Orphan Drug for NF
In February AstraZeneca and Merck announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) for selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1). Research and clinical trials on selumetinib and other MEK inhibitors have been possible in great part due to the lobbying work of…
Read MoreNF Midwest Funds NF2 Grant to MGH Researcher
NF Midwest, in collaboration with NF Northeast, has awarded Dr. Vijaya Ramesh from Massachusetts General Hospital, a grant for research in neurofibromatosis type 2 to test the combination of a novel drug with an mTORC1/mTORC2 inhibitor in meningioma and schwannoma cell systems. This study directly stems from the very encouraging large amount of data generated by Synodos…
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