Results 51 to 60 of about 2,408,361 (302)
Scientific ReportsImmunoglobulin light chain amyloidosis (AL amyloidosis) is among the most common forms of systemic amyloidosis. Using electronic health records (EHR) data from the United States, we aimed to estimate the incidence and prevalence of AL amyloidosis over ...
Pedro A. Laires +7 more
doaj +1 more source
Giant intra-abdominal hydatid cysts with multivisceral locations [PDF]
, 2008 The disseminated intra-peritoneal hydatid disease is a very rare finding. A case of disseminated intra abdominal hydatid disease is presented along with a review of literature and various therapeutic ...
Kaushal, Satinder Singh +5 more
core +1 more source
Pediatric Blood &Cancer, EarlyView.ABSTRACT Arteriovenous malformations (AVMs) are rare, high‐flow, vascular anomalies that can occur either sporadically or as part of a genetic syndrome. AVMs can progress with serious morbidity and even mortality if left unchecked. Sirolimus is an mTOR inhibitor that is effective in low‐flow vascular malformations; however, its role in AVMs is unclear.
Will Swansson +3 more
wiley +1 more source
Clinical and Translational ScienceProperdin is an endogenous positive regulator of the complement alternative pathway (AP). Tarperprumig (ALXN1820), a novel humanized bispecific antibody, binds properdin and albumin and is being developed to treat complement‐mediated diseases. This phase
Avner Sandhu +8 more
doaj +1 more source
Outcomes of Live Virus Vaccination in Patients With Vascular Anomalies Being Treated With Sirolimus
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background Live vaccination in patients with vascular anomalies (VA) receiving sirolimus remains controversial due to immunosuppressive effects and theoretical risks. Procedure This single‐center retrospective study included patients with VA less than 4 years old at the start of sirolimus therapy who were incompletely vaccinated.
Svatava Merkle +5 more
wiley +1 more source
Summary of Research: Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis
Neurology and Therapy, 2023 This article provides a summary of a previously published paper: Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis. The paper reported the results of the CHAMPION-MG trial which investigated the drug ravulizumab in the rare ...
Tuan Vu +9 more
doaj +1 more source
An Unusual Case of Tertiary Syphilis Behaving Like Tongue Squamous Cell Carcinoma. [PDF]
, 2018 Syphilis may present with a myriad of oral manifestations in the primary, secondary, and tertiary stages, and may be confused with malignancy. Despite a rise in the incidence of syphilis, tertiary syphilis is exceedingly rare.
Farwell, D Gregory +2 more
core +2 more sources
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background The management of clinically apparent single lesions or oligofocal nephroblastomatosis, a facultative precursor of nephroblastoma, remains debated. Methods We retrospectively analyzed 37 patients with clinically apparent single or oligofocal nephroblastomatosis (two to three lesions per kidney) among 2347 patients registered between
Nils Welter +17 more
wiley +1 more source
Treatment responses to antiangiogenetic therapy and chemotherapy in nonsecreting paraganglioma (PGL4) of urinary bladder with SDHB mutation: a case report [PDF]
, 2018 Paraganglioma (PGL) is a rare neuroendocrine tumor. Currently, the malignancy is defined as the presence of metastatic spread at presentation or during follow-up.
Aschelter, Am +10 more
core +1 more source
Germline TP53 Mutations Causing Diamond–Blackfan Anemia: A French Report
Pediatric Blood &Cancer, EarlyView.ABSTRACT Diamond–Blackfan anemia is a rare congenital erythroblastopenia typically caused by mutations in ribosomal protein genes. Recently, gain‐of‐function mutations in TP53 have been identified as a novel cause of Diamond–Blackfan anemia. We report two French patients who both harbored a heterozygous TP53 deletion (NM_000546.5: c.1077delA; p ...
Rafael Moisan +6 more
wiley +1 more source