Results 141 to 150 of about 45,763 (247)

Scurvy: A Rare Disease or a Rare Diagnosis?

open access: yesJournal of Emergencies, Trauma, and Shock
Introduction: Scurvy, the disease state caused by ascorbic acid deficiency, was once an extremely common disease but is now thought to be a rare disease in postmodern societies. Physicians are not trained to consider scurvy as a possible diagnosis in patients at risk; rather, it is considered a rare diagnosis to add to a ...
openaire   +3 more sources

Gastrointestinal Perforation as a Safety Concern Among Patients With Rheumatoid Arthritis Receiving JAK Inhibitor Therapy: A Systematic Review and Network Meta‐Analysis

open access: yesArthritis Care &Research, EarlyView.
Objective Gastrointestinal perforation (GIP) is a rare and life‐threatening safety concern associated with JAK inhibitors (JAKi). We aimed to review the evidence regarding the risk of GIP associated with the use of JAKi in patients with rheumatoid arthritis (RA) using a systematic review and network meta‐analysis approach.
Thipsukhon Sathapanasiri   +7 more
wiley   +1 more source

Development of a personalized visualization and analysis tool to improve clinical care in complex multisystem diseases with application to scleroderma

open access: yesArthritis Care &Research, Accepted Article.
Background In complex diseases, it is challenging to assess a patient's disease state, trajectory, treatment exposures, and risk of multiple outcomes simultaneously, efficiently and at the point of care. Methods We developed an interactive patient‐level data visualization and analysis tool (VAT) that automates illustration of a scleroderma patient's ...
Ji Soo Kim   +18 more
wiley   +1 more source

Microphysiological Glomerular Filtration Barriers: Current Insights, Innovations, and Future Applications

open access: yesAdvanced Biology, EarlyView.
The glomerular filtration barrier (GFB) is the first step of blood filtration by the kidneys. The concerning increase of kidney diseases makes the development of new models essential. In this context, microphysiological glomerular filtration barriers focus on closely reproducing the physiological architecture of the in vivo GFB: podocytes, glomerular ...
Manon Miran   +5 more
wiley   +1 more source

Rare disease surveillance: An international perspective [PDF]

open access: bronze, 2001
Elizabeth Elliott   +5 more
openalex   +1 more source

Investigating the Cellular Effects of GALC Dosing in Enzyme Replacement Therapy for Krabbe Disease Supports the Role of Nanomedicine

open access: yesAdvanced Biology, EarlyView.
A detailed workflow for recombinant GALC production and characterization is presented to support enzyme replacement therapy for Krabbe disease. In vitro assays demonstrate that physiological GALC doses restore enzymatic activity and autophagic flux without affecting cell viability, whereas higher doses impair autophagy and reduce viability.
Ambra Del Grosso   +5 more
wiley   +1 more source

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