Analysis of the Situation of Unmet Health Care Need Among Extremely Rare Disease of Gaucher [PDF]
, 2020 Xinye Qi +9 more
openalex +1 more source
PCR225 Predictors of Health-Related Quality of Life across Rare Diseases: An Online Survey [PDF]
, 2023 Agam K. Rao +4 more
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Molecular Oncology, EarlyView.Liquid biopsy enables minimally invasive, real‐time molecular profiling through analysis of circulating biomarkers in biological fluids. This Perspective highlights the importance of training pathologists through integrative educational programs, such as the European Masters in Molecular Pathology, to ensure effective and equitable implementation of ...
Marius Ilié +13 more
wiley +1 more source
Associations with Single Rare Heterozygous NLRP7 Variants for Chinese Patients with Sporadic Gestational Trophoblastic Diseases [PDF]
, 2020 Peiwen Zhang +13 more
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Effective therapeutic targeting of CTNNB1‐mutant hepatoblastoma with WNTinib
Molecular Oncology, EarlyView.WNTinib, a Wnt/CTNNB1 inhibitor, was tested in hepatoblastoma (HB) experimental models. It delayed tumor growth and improved survival in CTNNB1‐mutant in vivo models. In organoids, WNTinib outperformed cisplatin and showed enhanced efficacy in combination therapy, supporting its potential as a targeted treatment for CTNNB1‐mutated HB.
Ugne Balaseviciute +17 more
wiley +1 more source
Urgent Support and Aid Needed for Ukrainian Patients with Rare Diseases and their Families [PDF]
, 2022 Michael Wilbur, Anna Kole
openalex +1 more source
Molecular Oncology, EarlyView.The cancer problem is increasing globally with projections up to the year 2050 showing unfavourable outcomes in terms of incidence and cancer‐related deaths. The main challenges are prevention, improved therapeutics resulting in increased cure rates and enhanced health‐related quality of life.
Ulrik Ringborg +43 more
wiley +1 more source
A very rare case of encephalopathy in a patient with end-stage renal disease: contrast agent, ioversol [PDF]
, 2010 Rüya Özelsancak +5 more
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Therapeutic strategies for MMAE‐resistant bladder cancer through DPP4 inhibition
Molecular Oncology, EarlyView.We established monomethyl auristatin E (MMAE)‐resistant bladder cancer (BC) cell lines by exposure to progressively increasing concentrations of MMAE in vitro. RNA sequencing showed DPP4 expression was increased in MMAE‐resistant BC cells. Both si‐DPP4 and the DPP4 inhibitor sitagliptin suppressed the viability of MMAE‐resistant BC cells.
Gang Li +10 more
wiley +1 more source
Lafora Disease Masquerading as Hepatic Dysfunction [PDF]
, 2018 Lafora disease is fatal intractable progressive myoclonic epilepsy. It is frequently characterized by epileptic seizures, difficulty walking, muscle spasms, and dementia in late childhood or adolescence.
Abdullah, Hafez Mohammad A. +6 more
core +1 more source