Results 51 to 60 of about 7,599,301 (308)
STAR Protocols, 2022 Summary: Here, we present a protocol to image a fluorescent-labeled intraflagellar transport (IFT) component in Caenorhabditis elegans with fluorescence microscopy, including steps of sample preparations, in vivo live-cell imaging, and post-microscopy ...
Merve G. Turan +5 more
doaj +1 more source
Critical care (London, England), 2007 Thrombocytopenia is a common finding in critically ill patients. Heparin-induced thrombocytopenia is an infrequent cause of a low platelet count. Intensivists should use the diagnostic classification system developed by the International Society on Thrombosis and Haemostasis to diagnose heparin-induced thrombocytopenia.
openaire +2 more sources
The Challenge of Rare Diseases [PDF]
Chest, 2018 Rare diseases pose particular challenges to patients who are affected, to the clinicians who care for them, and to the investigators who study their conditions. Although individually uncommon, rare diseases are common in the aggregate, with approximately 7,000 described rare diseases affecting 25 to 30 million US adults.
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Ewing Sarcoma in Infants and Children Under 2 Years of Age: A French Retrospective Study
Pediatric Blood &Cancer, EarlyView.ABSTRACT Ewing sarcoma, the second most common primary bone cancer in children, requires intensive treatment that may lead to significant long‐term sequelae, particularly in infants. We retrospectively analyzed data from 1621 French patients treated between 1988 and 2015 within the EW88/93/97 or EE99 trials, focusing on 17 infants diagnosed before 24 ...
Elodie Verdier +18 more
wiley +1 more source
Improving lives optimising resources: a vision for the UK Rare Disease Strategy
, 2011 This report was developed over 18 months and over 1000 stakeholders and organisations from a wide range of backgrounds contributed to its production. We believe this to be the most comprehensive and wide-ranging review of services for patients with rare ...
Lucas, Jane S., Rare Diseaese UK
core +1 more source
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
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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
Collecting rare diseases [PDF]
F1000Research, 2014 This editorial introduces the F1000Research rare disease collection. It is common knowledge that for new treatments to be successful there has to be a partnership between the many interested parties such as the patient, advocate, disease foundations, the academic scientists, venture funding organizations, biotech companies, pharmaceutical companies ...
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Pediatric Blood &Cancer, EarlyView.ABSTRACT Background Pediatric thromboembolism is increasingly encountered in critical care. Systemic thrombolysis with tissue plasminogen activator (tPA) facilitates vessel or valve patency, yet pediatric‐specific protocols remain undefined, and safety concerns persist. Objective To evaluate the efficacy and safety of a tailored, prolonged systemic tPA
Eran Shostak +5 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
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