Results 51 to 60 of about 9,174,709 (287)

ANK1 and EPB41 Variants and The Risk of Glucocorticoid‐Induced Osteonecrosis

Arthritis &Rheumatology, EarlyView.
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, Xianmin Zhu, Zhongzhong Chen, Wei Chen, Hussam Alkaissi, Zeng Zhang, Yun Gao, Junhui Yin, Xiangtian Yu, Qiong Fu, Youshui Gao, Zhenzhong Zhu, Qianying Cai, Pengwei Zhang, Weibin Chen, Xin Zhao, Xiaojin Wu, Karel Pacak, Dongxu Jin, Chunde Bao, Zhengping Zhuang, Changqing Zhang   +21 more
wiley   +1 more source

Precision medicine in paediatrics: Progress and priorities

British Journal of Clinical Pharmacology, EarlyView.
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, Claire Booth, Joseph F. Standing, Charlotte I. S. Barker   +3 more
wiley   +1 more source

Clinical manifestations of sickle cell disease in Africa and its association with foetal haemoglobin parameters

Communications Medicine
Background Prevalence of sickle cell disease (SCD) across African countries ranges between 1–3% and contributes up to 7–16% of under-five mortality.
Evans Xorse Amuzu, Florence Urio, Elliot Eli Dogbe, Peter Ponsian, Suraj Yawnumah Abubakar, Chinedu Okeke, Olasinbo Olumuyiwa Balogun, Petronilla Jean Ozumba, Alex Osei-Akoto, Vivian Paintsil, Obiageli Nnodu, Emmanuel Balandya, Julie Makani, Madu Anazoeze, Daniel Ansong, Siana Nkya   +15 more
doaj   +1 more source

Evaluating the (comparative) safety profile of the novel oral polio vaccine type 2 using individual case safety reports in VigiBase

British Journal of Clinical Pharmacology, EarlyView.
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, Malede Mequanent Sisay, Leonardo Roque‐Pereira, Christine Leopold, Patrick C. Souverein, Aukje K. Mantel‐Teeuwisse, Marie L. De Bruin   +6 more
wiley   +1 more source

Ceftriaxone‐associated immune haemolytic anaemia: A patient‐level systematic review

British Journal of Clinical Pharmacology, EarlyView.
Abstract Ceftriaxone can cause acute haemolysis. Its clinical presentation, risk factors, and outcomes remain incompletely characterized. We conducted a systematic review of published cases with patient‐level data, following international guidelines and with registration in PROSPERO (CRD420261338709). Reports were identified through three bibliographic
Sonja Miotti, Giada Wyttenbach, Mario G. Bianchetti, Camilla Lavagno, Pietro Camozzi, Gregorio P. Milani, Renato Gualtieri, Sebastiano A. G. Lava, Pietro B. Faré, Simone Janett   +9 more
wiley   +1 more source

Cell and Gene Therapies Manufacturing Challenges and Integrated Good Manufacturing Practices Solutions: A Lifecycle Perspective

Biotechnology and Bioengineering, EarlyView.
Lifecycle perspective on cell and gene therapy manufacturing challenges and enabling GMP solutions. ABSTRACT Cell and gene therapies (CGTs) are revolutionizing modern medicine; however, making these advanced medicines scalable and readily available to commercial manufacturers worldwide is a major challenge. The number of approved CGT products continues
Rajath Samaga, Udisha Singh, Sachin Dattram Pawar   +2 more
wiley   +1 more source

SICKLE CELL DISEASE AND PREGNANCY

Mediterranean Journal of Hematology and Infectious Diseases, 2019
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, Pooja Lodha, Roshan Colah, Prachi Atmapoojya, Prachi Atmapoojya   +4 more
openaire   +4 more sources

Status and future of recombinant adeno‐associated virus vector manufacturing

Biotechnology Progress, EarlyView.
Abstract Sixty years of adeno‐associated virus (AAV) research illustrates a trajectory marked by basic science exploration, iterative innovation, persistent challenges, a number of clinical setbacks, as well as commercial therapeutic triumphs. This continual evolution has led to recombinant AAV (rAAV) becoming a cornerstone of modern gene therapy ...
Frank Agbogbo, David Dismuke
wiley   +1 more source

What you should know about sickle cell trait [PDF]

Sickle cell trait (SCT) is not a mild form of sickle cell disease. Having SCT simply means that a person carries a single gene for sickle cell disease (SCD) and can pass this gene along to their children.

core   +2 more sources

Unveiling Extramedullary Hematopoiesis: A Case Report Highlighting the Causes, Symptoms, and Management Strategies

Thalassemia Reports
Extramedullary hematopoiesis (EMH) serves as a compensatory mechanism in chronic hemolytic anemias, such as thalassemia, and can result in spinal cord compression.
Konstantinos Manganas, Aikaterini Xydaki, Angeliki Kotsiafti, Olympia Papakonstantinou, Sophia Delicou   +4 more
doaj   +1 more source