Results 31 to 40 of about 118,583,499 (297)

Establishment and Evolution of China National Hemophilia Registry

罕见病研究, 2022
Hemophilia is an inherited bleeding disorder and a type of rare disease that is hereditary, lifelong and disabling. The establishment of a National Hemophilia Registry is foundational to treating hemophilia.
XUE Feng, YANG Renchi
doaj   +1 more source

Cerebral Small Vessel Disease in Immune‐Mediated Thrombotic Thrombocytopenic Purpura Patients During the Acute Phase and Disease Remission

American Journal of Hematology, EarlyView.
ABSTRACT Immune‐mediated thrombotic thrombocytopenic purpura (iTTP) is a life‐threatening thrombotic microangiopathy characterized by acute neurological manifestations and long‐term sequelae related to microvascular brain injury. Cerebral small vessel disease (cSVD), detectable on MRI through lacunes, microbleeds, white matter hyperintensities, and ...
Addolorata Truma, Francesco Maria Lo Russo, Giorgio Conte, Ilaria Mancini, Andrea Artoni, Juri Alessandro Giannotta, Barbara Ferrari, Pasquale Agosti, Maria Abbattista, Matteo Gagliardi, Eleonora Piccin, Marco Stroppi, Fabio Maria Triulzi, Flora Peyvandi   +13 more
wiley   +1 more source

Venous Thromboembolism Prophylaxis in Hemophilic Patients Undergoing Total Hip or Knee Arthroplasty: Insights from a Single-Center Experience

Medicina
Background and Objectives: Total hip replacement and total knee arthroplasty carry a high risk of postoperative venous thromboembolism (VTE); therefore, anticoagulation prophylaxis is recommended in these patients.
Oana-Viola Badulescu, Paul-Dan Sirbu, Manuela Ciocoiu, Maria Cristina Vladeanu, Carmen Elena Plesoianu, Andrei Bojan, Dan Iliescu-Halitchi, Razvan Tudor, Bogdan Huzum, Mihnea-Theodor Sirbu, Norin Forna, Gheorghe Sofron, Wilhelm Friedl, Iris Bararu-Bojan   +13 more
doaj   +1 more source

Bleeding Patterns among Severe Hemophilia A and B Patients in West Java

Althea Medical Journal, 2020
Background: The clinical manifestations of hemophilia A (HA) and hemophilia B (HB)are quite similar; however, the bleeding characteristics of these two hemophilia types have been reported to be different. This study aimed to explore the bleeding patterns
Muhammad Mufakkirul Islam, Susi Susanah, Amaylia Oehadian   +2 more
doaj   +1 more source

Engineering Biology Beyond Single Genes: Advances and Challenges in Multiplex Genome Editing

Animal Research and One Health, EarlyView.
Multiplex genome editing is transforming genome engineering from single‐gene perturbation to network‐level control, yet its broader application remains limited by challenges in gRNA array engineering, delivery technologies, and safety management. Emerging AI‐driven approaches are accelerating guide RNA design and CRISPR effector optimization for ...
Linli Wang, Yongbin Liu, Hongbing Han
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

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

Clinical, instrumental, serological and histological findings suggest that hemophilia B may be less severe than hemophilia A

Haematologica, 2016
Recent evidence suggests that patients with severe hemophilia B may have a less severe disease compared to severe hemophilia A. To investigate clinical, radiological, laboratory and histological differences in the arthropathy of severe hemophilia A and ...
Daniela Melchiorre, Silvia Linari, Mirko Manetti, Eloisa Romano, Francesco Sofi, Marco Matucci-Cerinic, Christian Carulli, Massimo Innocenti, Lidia Ibba-Manneschi, Giancarlo Castaman   +9 more
doaj   +1 more source

Gene therapy for hemophilia B

Bleeding, Thrombosis and Vascular Biology
Dear Editor, I wish to report that the first gene therapy procedure in Italy for the treatment of severe or moderately severe hemophilia B has been successfully administered at the Policlinico of Milan....
Pier Mannuccio Mannucci
doaj   +1 more source

Hemophilia [PDF]

, 2012
This book demonstrates the great efforts aimed at further improving the care of the hemophilia, which may bring further improvement in the quality of life of hemophilia persons and their ...

core   +1 more source