Results 71 to 80 of about 27,651 (232)

Evolving Real‐World Data and Evidence Use for New Drugs and Regenerative Medical Products Approvals in Japan—An Analysis of the 6‐Year Trend

Clinical Pharmacology &Therapeutics, Volume 118, Issue 6, Page 1405-1421, December 2025.
Several regulatory initiatives have been made to clarify the acceptability and requirements of real‐world data and real‐world evidence (RWD/E) for the benefit/risk assessment of new medical products in Japan. The objectives of this review were to characterize the use of RWD/E in regulatory applications of new medical products and to describe the ...
Suguru Okami, Kako Shimotsumagari, Yasunari Sadatsuki   +2 more
wiley   +1 more source

Healthcare professionals' perspectives on supporting individuals with NF1 during pregnancy and decision‐making processes

Journal of Genetic Counseling, Volume 34, Issue 6, December 2025.
Abstract Neurofibromatosis type 1 (NF1) is an autosomal dominant genetic condition characterized by highly variable presentation, making reproductive decision‐making and pregnancy care particularly complex. While previous research has focused largely on clinical outcomes, little is known about how healthcare professionals (HCPs) provide care and ...
Gamze Kaplan, Debbie M. Smith, Ming Wai Wan, Emma Burkitt‐Wright, Shruti Garg   +4 more
wiley   +1 more source

Facial plexiform neurofibroma in a child with neurofibromatosis type I: A case report

Journal of Indian Society of Pedodontics and Preventive Dentistry, 2007
Plexiform neurofibroma is a non-circumscribed, thick, and irregular benign tumor of the peripheral nerve sheath. It is a virtually pathognomonic and often disabling feature of neurofibromatosis type I.
Patil K, Mahima V, Shetty S, Lahari K
doaj  

Ataluren‐Induced Functional Restoration of Neurofibromin in Fibroblasts From Neurofibromatosis Type 1 Patients With Nonsense Mutations

MedComm, Volume 6, Issue 12, December 2025.
The therapeutic potential of ataluren for NF1 nonsense mutations was assessed by a comprehensive evaluation of the RAS/MEK/ERK pathway activity in 22 NF1NS/+ patient‐derived fibroblasts. The whole‐transcriptomic profiles between ataluren responders and nonresponders, and further analysis and validation revealed novel biomarkers, AMPD3 and TGFBR3, for ...
Soyoung Kim, Hyosang Do, Sun Hee Heo, Minji Kang, Soojin Hwang, Dohyung Kim, Min‐Hoo Chang, Kyung Kim, Beom Hee Lee   +8 more
wiley   +1 more source

Cardiac overload resolved by resection of a large plexiform neurofibroma on both the buttocks and upper posterior thighs in a patient with neurofibromatosis type I: a case report

BMC Surgery, 2020
Background A large plexiform neurofibroma in patients with neurofibromatosis type I can be life threatening due to possible massive bleeding within the lesion.
Taro Mikami, Yuki Honma-Koretsune, Yui Tsunoda, Shintaro Kagimoto, Yuichiro Yabuki, Jiro Maegawa, Takashi Terauchi, Shintaro Nawata, Hiroyuki Kamide, Yoshinobu Ishiwata, Tabito Kino, Teruyasu Sugano   +11 more
doaj   +1 more source

Surgical Treatment and Complications of Deep-Seated Nodular Plexiform Neurofibromas Associated with Neurofibromatosis Type 1 [PDF]

, 2022
K. Ikuta, Yoshihiro Nishida, Tomohisa Sakai, Hiroshi Koike, Kan Ito, Hiroshi Urakawa, Shiro Imagama   +6 more
openalex   +1 more source

Nanodroplet Array Platform for Integrated Synthesis and Screening of MEK Inhibitors: a Miniaturized Approach to Early Drug Discovery

Angewandte Chemie International Edition, Volume 64, Issue 48, November 24, 2025.
We present a nanodroplet array platform that integrates solid‐phase synthesis, MALDI‐MS analysis, and cell‐based screening. Using nanoliter droplets, we synthesized and tested 325 potential MEK inhibitors and identified 46 active compounds. This miniaturized approach combines chemistry and biology on a single chip, reducing time, cost, and material use
Maximilian Seifermann, Julius Höpfner, Liana Bauer, Divya Varadharajan, Stefan Schmidt, Björn Fröhlich, Benjamin Wellenhofer, Charlotte Luchena, Carsten Hopf, Anna A. Popova, Pavel A. Levkin   +10 more
wiley   +1 more source

Multidimensional ultrasound and computed tomography imaging support in bleeding plexiform neurofibromatosis of the scalp: A case report and literature review

Indian Journal of Dermatology, 2015
Active bleeding in plexiform neurofibromatosis can be a life-threatening complication in neurofibromatosis type 1 (NF1). The prompt imaging support of 2D-3D ultrasound (US) and computed tomography (CT) during the active hemorrhage phase of cutaneous ...
Ximena Wortsman, Nelson Lobos, Rodrigo De la Parra, Laura Carreno   +3 more
doaj   +1 more source

A Ten‐Step Surgical Approach to Radical Excision of a Primary Ischiorectal Fossa Tumor With IGAP V‐Y Flap Reconstruction—A Case Report of a Rare Myxofibrosarcoma

Clinical Case Reports, Volume 13, Issue 11, November 2025.
ABSTRACT This case report presents a rare case of ischiorectal fossa myxofibrosarcoma, which was successfully excised with a clear margin and full preservation of the patient's pudendal nerve function. We present our surgical technique in 10 steps, describing the surgical excision and reconstructive surgery with the inferior gluteal artery perforator V‐
Amy Whitmore, Sabina Ioana Nistor, Muhammad Ather Siddiqi, Roman Mykula, Hooman Soleymani majd   +4 more
wiley   +1 more source

Diagnosis and Management of an Isolated Pediatric Plexiform Neurofibroma Involving the Hepatic and Celiac Plexus Using Multimodality Approach: Problem Solving with Diffusion-Weighted Magnetic Resonance Imaging

European Journal of Pediatric Surgery Reports, 2013
Plexiform neurofibroma with involvement of the gastrointestinal tract is a very rare entity in children. Here, we present a rather unique case of a 9-year-old boy with no clinical signs or features of neurofibromatosis type 1.
Merel M. Scheurkogel, June Koshy, Kenneth J. Cohen, Thierry A. G. M. Huisman, Thangamadhan Bosemani   +4 more
doaj   +1 more source