Target Product Profile for Cutaneous Neurofibromas: Clinical Trials to Prevent, Arrest, or Regress Cutaneous Neurofibromas
العنوان: | Target Product Profile for Cutaneous Neurofibromas: Clinical Trials to Prevent, Arrest, or Regress Cutaneous Neurofibromas |
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المؤلفون: | Ly, Ina, Romo, Carlos, Gottesman, Sally, Kelly, Kristen M, Kornacki, Deanna, York, Zachary, Lee, Sang Y, Rhodes, Steven D, Staedtke, Verena, Steensma, Matthew R, Blakeley, Jaishri O, Wolkenstein, Pierre |
المصدر: | The Journal of investigative dermatology, vol 143, iss 8 |
بيانات النشر: | eScholarship, University of California, 2023. |
سنة النشر: | 2023 |
مصطلحات موضوعية: | Rare Diseases, Clinical Research, Dermatology & Venereal Diseases, Clinical Trials and Supportive Activities, Clinical Sciences, Oncology and Carcinogenesis, Cancer |
الوصف: | Cutaneous neurofibromas (cNFs) are benign tumors of the skin that affect >95% of adults with neurofibromatosis type 1. Despite their benign histology, cNFs can significantly impact QOL due to disfigurement, pain, and pruritus. There are no approved therapies for cNFs. Existing treatments are limited to surgery or laser-based treatments that have had mixed success and cannot be readily applied to a large number of tumors. We review cNF treatment options that are currently available and under investigation, discuss the regulatory considerations specific to cNFs, and propose strategies to improve cNF clinical trial design and standardize clinical trial endpoints. |
وصف الملف: | application/pdf |
الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=od_______325::223e9ddbf84dd2c4a3b352af38f3d7feTest https://escholarship.org/uc/item/2j02s0htTest |
حقوق: | OPEN |
رقم الانضمام: | edsair.od.......325..223e9ddbf84dd2c4a3b352af38f3d7fe |
قاعدة البيانات: | OpenAIRE |