Results 71 to 80 of about 7,998,845 (308)
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
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Therapeutic Apheresis and Dialysis, EarlyView.ABSTRACT Background Japan has one of the highest dialysis prevalence rates worldwide and a shrinking, aging population. Whether dialysis burden has entered a sustained post‐peak phase or whether recent declines partly reflect pandemic‐related disruptions remains uncertain.
Hatice Şahin +2 more
wiley +1 more source
CPT: Pharmacometrics & Systems PharmacologyDanicopan is a first‐in‐class orally administered complement factor D inhibitor, approved as an add‐on therapy to ravulizumab or eculizumab for the treatment of clinically significant extravascular hemolysis in patients with paroxysmal nocturnal ...
Jun Chen +4 more
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Organoids in pediatric cancer research
FEBS Letters, EarlyView.Organoid technology has revolutionized cancer research, yet its application in pediatric oncology remains limited. Recent advances have enabled the development of pediatric tumor organoids, offering new insights into disease biology, treatment response, and interactions with the tumor microenvironment.
Carla Ríos Arceo, Jarno Drost
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Scientific ReportsClinically, α-thalassemia is stratified into mild, intermediate, and severe forms, differentiated by the degree of anemia severity. Specifically, severe α-thalassemia manifests in homozygous individuals, characterized by a profound α globin deficit.
Xin Xu, Wencheng Fu, Wenrui Ye
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MicroorganismsOne of the primary challenges in working with adeno-associated virus (AAV) lies in the inherent instability of its inverted terminal repeats (ITRs), which play vital roles in AAV replication, encapsidation, and genome integration.
Yinxing Chen +11 more
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TH Open, 2017 Patients with acute coronary syndrome with diabetes mellitus (DM) exhibit an impaired platelet inhibitory response to clopidogrel which is only partially understood. DM was induced by the administration of streptozotocin (STZ) to 9-week-old mice.
A. Sugidachi +5 more
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An Easy Guide to Rare Diseases in Ireland: A Resource for the Media and the General Public
, 2020 This ‘Easy Guide’ has been produced to increase awareness about the challenges of living with a rare disease and to highlight the priorities that need to be included in the forthcoming National Plan for Rare Diseases (NPRD) in Ireland. The aims of this
Task Force on Rare Diseases
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From mice to humans—divergent strategies for intestinal homeostasis and regeneration
FEBS Letters, EarlyView.Recent advances such as organoid genome editing, xenotransplantation, imaging, and whole‐genome sequencing have enabled direct studies of human intestinal stem cells (ISCs). These studies reveal species‐specific features, including slower ISC proliferation, distinct injury responses, slower somatic mutation accumulation in humans, and an inverse ...
Keiko Ishikawa +2 more
wiley +1 more source
Investigating transcription factor dynamics in health and disease using FRAP
FEBS Letters, EarlyView.FRAP analysis of GFP‐tagged transcription factors reveals how molecular mobility and target engagement change in response to drug treatment. By combining live‐cell imaging, quantitative model fitting, and statistical analysis, this approach uncovers transcription factor dynamics linked to disease mechanisms, providing a powerful framework for ...
Kannan Govindaraj +3 more
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