Results 51 to 60 of about 88,496 (299)
ANK1 and EPB41 Variants and the Risk of Steroid‐Induced Osteonecrosis
Objective Steroid‐induced osteonecrosis of the femoral head (SONFH) is a refractory skeletal disorder influenced by genetic and environmental factors. However, conclusive pathogenic genetic evidence remains elusive due to the limited exploration of rare damaging variants. In this study, we aimed to identify rare variants associated with SONFH.
Shengbao Chen +21 more
wiley +1 more source
Asthma in Sickle Cell Disease [PDF]
In recent years, evidence has increased that asthma predisposes to complications of sickle cell disease (SCD), such as pain crises, acute chest syndrome, pulmonary hypertension, and stroke, and is associated with increased mortality. An obstructive pattern of pulmonary function, along with a higher-than-expected prevalence of airway hyper ...
Manisha Newaskar +2 more
openaire +3 more sources
Precision medicine in paediatrics: Progress and priorities
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain +3 more
wiley +1 more source
SICKLE CELL DISEASE AND PREGNANCY
Abstract Sickle Cell Disease (SCD) is a group of inherited single-gene autosomal recessive disorders caused by the ‘sickle’ gene, which affects haemoglobin structure. Sickle cell anemia is the most common hemoglobinopathy worldwide. The burden of sickle cell disease in pregnancy has been exponentially increasing with more number of women reaching ...
Dipty Jain +4 more
openaire +4 more sources
Aim Novel oral polio vaccine type 2 (nOPV2) was used under the WHO emergency use listing for circulating vaccine‐derived polio virus (cVDPV) outbreaks from 2021 to 2023. We assessed nOPV2 adverse events following immunization (AEFIs) and compared its safety profile to other vaccines using VigiBase.
Comfort Kunak Ogar +6 more
wiley +1 more source
Sickle Cell Disease in Children [PDF]
Early identification of infants with sickle cell disease (SCD) by newborn screening, now universal in all 50 states in the US, has improved survival, mainly by preventing overwhelming sepsis with the early use of prophylactic penicillin. Routine transcranial Doppler screening with the institution of chronic transfusion decreases the risk of stroke from
Emily Riehm, Meier, Jeffery L, Miller
openaire +2 more sources
Aim A prior non‐randomized study suggests that potassium supplementation may improve survival among furosemide initiators, and a randomized trial suggests that salt substitutes containing potassium might lower stroke risk. We conducted a retrospective cohort study using health‐care data to confirm or refute these associations among new users of ...
Thanh Phuong Pham Nguyen +8 more
wiley +1 more source
Thrombosis and Sickle Cell Disease
Sickle cell disease (SCD) is characterized by the presence of sickle hemoglobin, which has the unique property of polymerizing when deoxygenated. The pathophysiology of acute and chronic clinical manifestations of SCD have shown the central role of dense, dehydrated red cells in acute and chronic clinical manifestations of this pathology.
DE FRANCESCHI, Lucia +2 more
openaire +2 more sources
Neuroimaging Biomarkers in Paediatric Sickle Cell Disease
Sickle Cell Disease (SCD) is a collection of genetic haemoglobinopathies, the most common and severe being homozygous sickle cell anaemia. In the UK, it has been estimated that 1 in 2000 children are born with SCD. The disease is characterised by chronic
Kawadler, JM
core
Advanced therapy medicinal products (ATMPs) – An analysis of the global regulatory status
Abstract Background A rapid progress in the development of advanced therapy medicinal products (ATMPs) has taken place over the past decades. The ATMPs hold great potential but face challenges in the clinical development and the regulatory process.
Allan Cramer +4 more
wiley +1 more source

