Results 141 to 150 of about 13,095,395 (361)
Can a regulatory instrument promote research and development into neglected diseases? A Policy Delphi Survey [PDF]
, 2012 Fehr A. Can a regulatory instrument promote research and development into neglected diseases? A Policy Delphi Survey. Bielefeld: University of Bielefeld; 2012.Background Rare diseases and neglected diseases are characterized by deficits in drug research ...
Fehr, Angela
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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
A systematic review of studies that estimated the burden of chronic non-communicable rare diseases using disability-adjusted life years [PDF]
Background: Initiatives aiming to assess the impact of rare diseases on population health might be hampered due to the complexity of disability-adjusted life years (DALYs) estimation. This study aimed to give insight into the epidemiological data sources
Kolkhir, P. V. +8 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
wiley +1 more source
A population-based registry as a source of health indicators for rare diseases: the ten-year experience of the Veneto Region's rare diseases registry [PDF]
, 2014 Background: Although rare diseases have become a major public health issue, there is a paucity of population-based data on rare diseases. The aim of this epidemiological study was to provide descriptive figures referring to a sizable group of unrelated ...
FACCHIN, PAOLA +9 more
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FEBS Letters, EarlyView.We identified a systemic, progressive loss of protein S‐glutathionylation—detected by nonreducing western blotting—alongside dysregulation of glutathione‐cycle enzymes in both neuronal and peripheral tissues of Taiwanese SMA mice. These alterations were partially rescued by SMN antisense oligonucleotide therapy, revealing persistent redox imbalance as ...
Sofia Vrettou, Brunhilde Wirth
wiley +1 more source
Consideration on " Patient-Centered" Drug Development for Rare Diseases [PDF]
The unmet clinical needs of patients with rare diseases persist. Many rare diseases lack effective treatments, and drug development for rare diseases faces greater challenges than that for common multiple diseases.
AI Xing +3 more
core +1 more source
Chronic pain and fatigue in multiple osteochondroma and Ollier disease, a systematic review
BMC Musculoskeletal DisordersIntroduction Multiple osteochondromas (MO) and Ollier disease are characterized by multiple tumours in the skeletal bones. They lead to bone deformity, restricted joint motion, interference with skeletal growth and increased risk of malignant ...
Ariane Kwiet +2 more
doaj +1 more source
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