Results 71 to 80 of about 3,491 (198)
International Journal of Rheumatic Diseases, Volume 29, Issue 4, April 2026.ABSTRACT Objective Acute anterior uveitis (AAU) is the most common extra‐musculoskeletal manifestation of axial spondyloarthritis (axSpA). This study compared the effect of the tumor necrosis factor inhibitor (TNFi) certolizumab pegol (CZP) with standard non‐biologic care, on AAU flare rate in patients with axSpA and high risk of recurrent uveitis ...
Nigil Haroon +8 more
wiley +1 more source
ASMD-FSMD Technique for Designing Digital Devices on FPGA
, 2020 Recently, there has been, on the one hand, an increase in the complexity of digital device designs and, on the other hand, an increase in the requirements for the development time and the reliability of the designs. One of the directions of solving this problem is developing new techniques for designing digital devices.This paper proposes a new ...
openaire +2 more sources
Acid sphingomyelinase deficiency in France: a retrospective survival study
Orphanet Journal of Rare DiseasesBackground Acid sphingomyelinase deficiency (ASMD) or Niemann–Pick disease types A, A/B, and B is a progressive, life-limiting, autosomal recessive disorder caused by sphingomyelin phosphodiesterase 1 (SMPD1) gene mutations.
Wladimir Mauhin +17 more
doaj +1 more source
Case Reports in Gastrointestinal Medicine, 2019 Introduction. Acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick Type A and Type B disease) is a rare, inherited metabolic disorder.
David Cassiman +3 more
doaj +1 more source
Dynamic Borrowing With a Bias‐Tolerance Cap in Augmented Randomized Controlled Trials
Statistics in Medicine, Volume 45, Issue 6-7, March 2026.ABSTRACT Although randomized controlled trials (RCTs) are the gold standard for evaluating the efficacy and safety of treatments, they are challenged by cost, duration, enrollment, or ethical concerns. A possible solution is to incorporate external control data as a hybrid control group, for which various statistical methods are available.
Kota Sawada +2 more
wiley +1 more source
Journal of Diabetes Investigation, Volume 17, Issue 3, Page 448-459, March 2026.ABSTRACT Objective To explore the association between serum fibrinogen (FIB) and clinicopathological features and renal prognosis in type 2 diabetic nephropathy (T2DN), and develop a web‐based dynamic model to predict renal progression. Methods This paper retrospectively enrolled 173 biopsy‐proven T2DN patients and stratified them by the optimal FIB ...
Lin Ning +5 more
wiley +1 more source
Sphingomyelin 16:0 is a therapeutic target for neuronal death in acid sphingomyelinase deficiency
Cell Death and Disease, 2023 Acid sphingomyelinase deficiency (ASMD) is a lysosomal storage disorder caused by mutations in the SMPD1 gene encoding for the acid sphingomyelinase (ASM). While intravenous infusion of recombinant ASM is an effective treatment for the peripheral disease,
Ángel Gaudioso +4 more
doaj +1 more source
Massive stars in sub-parsec rings around galactic centers
, 2005 We consider the structure of self-gravitating marginally stable accretion disks in galactic centers in which a small fraction of the disk mass has been converted into proto-stars.
Nayakshin, Sergei
core +3 more sources
Newborn Screening for Acid Sphingomyelinase Deficiency: Prevalence and Genotypic Findings in Italy
International Journal of Neonatal ScreeningAcid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder with a broad clinical spectrum. Early diagnosis and initiation of treatment are crucial for improving outcomes, yet the disease often goes undiagnosed due to its rarity and ...
Vincenza Gragnaniello +7 more
doaj +1 more source
Orphanet Journal of Rare DiseasesBackground Acid sphingomyelinase deficiency (ASMD) is a rare, progressive, potentially fatal lysosomal storage disease that exhibits a broad spectrum of clinical phenotypes.
Eugen Mengel +11 more
doaj +1 more source