Every person living with neurofibromatosis type 1 (NF1) has a story—and nearly all of those stories include cutaneous neurofibromas (cNFs). These benign tumors of the skin may not pose a life-threatening risk, but for the […]
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It can spawn tumors throughout the nervous system and bone deformities that riddle the whole body with pain. One of the most common rare diseases in the world, the genetic condition neurofibromatosis type 1, or NF1, can cause a wide spectrum of mild to severe symptoms—all stemming from a single mutation on a solitary gene: the NF1 gene on chromosome 17.
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NTAP’s innovative funding approach was featured in an article,”Pedal to the Metal” on Inside Hopkins, the Johns Hopkins Medicine daily publication highlighting exceptional and impactful programs. “How an innovative funding approach is advancing research and […]
Read MoreA forum to identify priorities and advance the field of neurofibromatosis type-1 (NF1) research Introduction to NTAP symposiaNTAP convenes leading researchers and innovators from across multiple fields and scientific backgrounds in regular symposia to establish […]
Read MoreThe Neurofibromatosis Therapeutic Acceleration Program (NTAP) at the Johns Hopkins University School of Medicine is proud to announce that Dr. Nicole Brossier, Dr. Suganth Suppiah, and Dr. Harish Vasudevan have been selected as the 2021 […]
Read MoreOn April 1, 2019, AstraZeneca (the maker of selumetinib) announced that the US Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for the MEK 1/2 inhibitor selumetinib for the treatment of paediatric patients aged three […]
Read MoreN Engl J Med 2020; 382:1430-1442 DOI: 10.1056/NEJMoa1912735 List of authors. Andrea M. Gross, M.D., Pamela L. Wolters, Ph.D., Eva Dombi, M.D., Andrea Baldwin, P.N.P., Patricia Whitcomb, R.N., Michael J. Fisher, M.D., Brian Weiss, M.D., […]
Read MorePatient derived tissues are a critical tool to support the ongoing research that improves our understanding of disease formation mechanisms, and helps drive the discovery of new therapeutics. As part of its commitment to providing […]
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