Ccl5 establishes an autocrine high-grade glioma growth regulatory circuit critical for mesenchymal glioblastoma survival [PDF]
Gutmann, David H +4 more
core +2 more sources
Usefulness of 18F-FDG-PET/CT in Evaluating a Brainstem Glioma in an Adult Patient with Neurofibromatosis Type 1 [PDF]
Giorgio Treglia +3 more
openalex +1 more source
Clinicopathological Characteristics and Outcomes of Genitourinary Rhabdomyosarcoma in Two Girls
ABSTRACT Rhabdomyosarcoma (RMS) is a soft tissue neoplasm accounting for about 8% of solid tumors in children. There are mainly four histologic subtypes of RMS: embryonal, alveolar, spindle cell/sclerosing, and pleomorphic. The genitourinary tract is the second most affected primary site of RMS. Genitourinary RMS is more common in young boys than girls.
George Evele +3 more
wiley +1 more source
Corpus callosum tumor as the presenting symptom of neurofibromatosis type 1 in a patient and literature review [PDF]
Introducción. La neurofibromatosis tipo 1 (NF1) es uno de los síndromes neurocutáneos más frecuentes y puede asociarse a tumores intracraneales en cualquier localización, pero excepcionalmente en el cuerpo calloso. Objetivos.
Pascual-Castroviejo, Ignacio +3 more
core +1 more source
Clinicopathological and Immunohistochemistry Study of a Long Survivor of Giant Cell Glioblastoma in a Patient With Neurofibromatosis 1: Case Report [PDF]
Noora Al-romaihi +4 more
openalex +1 more source
In the field of rare diseases—where traditional clinical trials are often impractical—real‐world data (RWD) have emerged as a scientifically valid alternative to support regulatory decision making. This study systematically evaluates the utilization of RWD in orphan drug approvals by the FDA Center for Drug Evaluation and Research (CDER) over the past ...
Minji Kim, Eunjin Hong
wiley +1 more source
Multiple glomus tumors and segmental neurofibromatosis: there are no coincidences [PDF]
Segmental neurofibromatosis is a rare subtype of neurofibromatosis type 1 (NF1). Glomus tumors are uncommon benign tumors.
Cabral, R, Santiago, F, Tellechea, O
core +1 more source
ABSTRACT Objective This systematic review and meta‐analysis aimed to assess the diagnostic yield of pathogenic or likely pathogenic (P/LP) single nucleotide variants (SNVs) using whole genome sequencing (WGS) in congenital heart disease (CHD). Methods A systematic search of three databases (2000–2024) was conducted, and two reviewers independently ...
Hiba J. Mustafa +7 more
wiley +1 more source
Novel drugs approved by the EMA, the FDA and the MHRA in 2025: A year in review
Abstract In the 2025 novel drug mini‐review, one can take a full measure of the ingenuity that underlies current drug design and development, despite the year's smaller harvest (46 novel drugs) compared to 2024 (53) and 2023 (70). 54% of the novel drugs are first‐in‐class (FIC).
Andreas Papapetropoulos +16 more
wiley +1 more source

