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btk inhibition suppresses neuroinflammation and neurodegeneration in amyotrophic lateral sclerosis
Original title: “BTK inhibition suppresses neuroinflammation and neurodegeneration in amyotrophic lateral sclerosis.”
Nature Reviews Gastroenterology & Hepatology - The ethics of unlicensed therapy use in primary biliary cholangitis: a counter view Greater transplant-free survival in patients receiving obeticholic acid for primary biliary cholangitis in a clinical trial setting compared to real-world external controls. Gastroenterology 163, 1630–1642.e3 (2022). ... Jones, D. E. J. et al. Primary biliary cholangitis drug evaluation and regulatory approval: where do we go from here? Hepatology 80, 1291–1300 (2024). ... Martins, A. et al. Comparative effectiveness of peroxisome proliferator–activated receptor agonists as second-line therapies for primary biliary cholangitis: a systematic review and network meta-analysis. Toronto Center for Liver Disease, University Health Network, Toronto, Ontario, Canada · Bettina E. Hansen · The Autoimmune and Rare Liver Disease Programme, Division of Gastroenterology and Hepatology, University Health Network, Toronto General Hospital, Toronto, Ontario, Canada The authors are all either officers or medical advisors to the PBC Foundation. All the authors other than R.M.-T. (who is a patient representative) are clinicians who are experienced investigators and have contributed to the development of licensed therapies in PBC. Table 1 Characteristics of the licensed and unlicensed second-line therapies currently used in primary biliary cholangitis ... Instant access to the full article PDF. ... Corpechot, C. et al. Bezafibrate for primary biliary cholangitis: time to act on the evidence. Nat. Rev. Gastroenterol. Hepatol. 22, 805–807 (2025). ... Corpechot, C. et al. A placebo-controlled trial of bezafibrate in primary biliary cholangitis.
Original title: “The ethics of unlicensed therapy use in primary biliary cholangitis: a counter view | Nature Reviews Gastroenterology & Hepatology”
bi allelic variants in fsd1l cause retinitis pigmentosa with or without neurological involvement
Original title: “Bi-allelic variants in FSD1L cause retinitis pigmentosa with or without neurological involvement.”
marked regression of calcinosis with canakinumab in hyperphosphatemic familial tumoral calcinosis
Original title: “Marked regression of calcinosis with canakinumab in hyperphosphatemic familial tumoral calcinosis.”
novel spliceosomopathy caused by de novo sf3b3 variants
Original title: “A novel spliceosomopathy caused by de novo SF3B3 variants.”
management of severe bone loss in patients at risk of medication related osteonecrosis of the jaw with microsurgery and guided bone regeneration a case study
Original title: “Management of severe bone loss in patients at risk of medication-related osteonecrosis of the jaw with microsurgery and guided bone regeneration: A case study.”
intraosseous sarcoidosis imitating peri implantitis a case study
Original title: “Intraosseous sarcoidosis imitating peri-implantitis: A case study.”
interferon driven tryptophan metabolism links inflammation and mental health in juvenile dermatomyositis
Original title: “Interferon-Driven Tryptophan Metabolism Links Inflammation and Mental Health in Juvenile Dermatomyositis.”
clinicopathological features and prognosis of patients with resectable has gaed a multicenter nested case control study from china
Original title: “Clinicopathological features and prognosis of patients with resectable HAS/GAED: A multicenter nested case-control study from China.”
monocyte macrophage samhd1 alleviates eamg by modulating the cgas sting pathway
Original title: “Monocyte-macrophage SAMHD1 alleviates EAMG by modulating the cGAS-STING pathway.”
simultaneous assessment of genetic and epigenetic contributions to the plasma lipid levels with respect to cardiovascular risk
Original title: “Simultaneous Assessment of Genetic and Epigenetic Contributions to the Plasma Lipid Levels with Respect to Cardiovascular Risk.”
acromegaly presenting with normal insulin like growth factor 1 levels in a patient with advanced liver cirrhosis
Original title: “Acromegaly presenting with normal insulin-like growth factor-1 levels in a patient with advanced liver cirrhosis.”
lecithin cholesterol acyltransferase deficiency with the finding of zebra bodies a diagnostic challenge in the context of suspected fabry disease
Original title: “Lecithin-cholesterol acyltransferase deficiency with the finding of "zebra bodies": A diagnostic challenge in the context of suspected Fabry disease.”
functional and structural outcomes in paediatric myelin oligodendrocyte glycoprotein antibody associated disease mogad a prospective study
Original title: “Functional and structural outcomes in paediatric myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD): a prospective study.”
tracing the path of non industry driven medicine development for rare diseases through regulatory interactions at the european medicines agency
Original title: “Tracing the path of non-industry-driven medicine development for rare diseases through regulatory interactions at the European Medicines Agency.”
mesenteric unicentric castleman s disease misdiagnosed as gastrointestinal neuroendocrine tumour
Original title: “Mesenteric unicentric Castleman's disease misdiagnosed as gastrointestinal neuroendocrine tumour.”
multiple opportunistic infections revealing erdheim chester disease an atypical presentation
Original title: “Multiple opportunistic infections revealing Erdheim-Chester disease: an atypical presentation.”
severe herpes simplex virus encephalitis complicated by atypical takotsubo cardiomyopathy with left ventricular systolic dysfunction
Original title: “Severe herpes simplex virus encephalitis complicated by atypical Takotsubo cardiomyopathy with left ventricular systolic dysfunction.”
artificial intelligence driven nano enhanced stem cell therapy for neurodegenerative diseases from rational design to clinical translation
Original title: “Artificial intelligence-driven nano-enhanced stem cell therapy for neurodegenerative diseases: from rational design to clinical translation.”
rationale and design of ultrasound applicability in the assessment of patients with fibrosing interstitial lung disease lord a research protocol of a prospective cohort study
Original title: “Rationale and design of uLtrasOund applicability in the assessment of patients with fibRosing interstitial lung Disease (LORD): a research protocol of a prospective cohort study.”