Results 71 to 80 of about 5,929,149 (180)

Bringing Gene Therapy Into Real World Clinical Practice

Haemophilia, EarlyView.
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach, Pratima Chowdary, Margareth C. Ozelo   +2 more
wiley   +1 more source

Compression therapy for treating stage I and II (Widmer) post-thrombotic syndrome.

, 2003
Item does not contain fulltextBACKGROUND: Post-thrombotic syndrome (PTS) is a long-term complication of deep vein thrombosis characterised by chronic complaints, swelling, and skin changes in the affected limb.
Sandbrink, M.W.C., Prins, M., Neumann, M.H.A.M., Kolbach, D.   +3 more
core   +1 more source

Rare Bleeding Disorders and Bleeding Disorder of Unknown Cause: Current Understanding and Recent Developments

Haemophilia, EarlyView.
ABSTRACT Rare bleeding disorders (RBDs) represent a diverse group of inherited conditions involving coagulation factors or platelets. These conditions, such as Glanzmann thrombasthenia (GT) or severe coagulation factor deficiencies, are uncommon. In contrast, bleeding disorder of unknown cause (BDUC) is a diagnosis of exclusion without an identifiable ...
Alessandro Casini, Robert Klamroth, Bauke Haisma, Dino Mehic, Saskia E. M. Schols   +4 more
wiley   +1 more source

Italian Patients Journey for Gene Therapy in Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient selection criteria, duration and variability of transgene expression, quality of life and long‐term safety.
Giovanni Di Minno, Gaia Spadarella, Alessandra Borchiellini, Anna Borrelli, Andrea Buzzi, Giancarlo Castaman, Michele Cecchi, Raimondo De Cristofaro, Vincenzo La Mura, Angelo Lupi, Giuseppe Malcangi, Maria Elisa Mancuso, Maria Francesca Mansueto, Cristina Santoro, Rita Carlotta Santoro, Luigi Piero Solimeno, Annarita Tagliaferri, Ezio Zanon, Isabella Garagiola, Flora Peyvandi   +19 more
wiley   +1 more source

Recombinant ADAMTS13 Ameliorates Liver Injury and Improves Hepatic Microcirculation in a Murine Acute‐on‐Chronic Liver Failure Model

Hepatology Research, EarlyView.
ABSTRACT Aim The therapeutic significance of recombinant a disintegrin‐like and metalloprotease with thrombospondin type 1 motif 13 (rADAMTS13) in acute‐on‐chronic liver failure (ACLF) remains unclear. We aimed to investigate the therapeutic effects of rADAMTS13 in a murine model of ACLF‐like liver injury.
Jun‐ichi Hanatani, Norihisa Nishimura, Kosuke Kaji, Masayoshi Takami, Hiroyuki Masuda, Hiroaki Takaya, Koh Kitagawa, Shinya Sato, Tadashi Namisaki, Hitoshi Yoshiji   +9 more
wiley   +1 more source

Thrombocytopenia in Pregnancy: A 5‐Year Analysis of Characteristics and Practices From a Tertiary Academic Center

International Journal of Laboratory Hematology, EarlyView.
ABSTRACT Background Moderate‐to‐severe thrombocytopenia (platelet count < 100 × 109/L) occurs in fewer than 1% of pregnancies, posing management challenges, particularly surrounding eligibility for neuraxial anesthesia. Although recent anesthesia guidelines recommend a platelet threshold ≥ 70 × 109/L, outcomes data applying these recommendations in ...
Mackenzie E. Lemieux, Ming Y. Lim
wiley   +1 more source

AI‐Enabled Automated Schistocyte Classification in Peripheral Blood for TMA Auxiliary Diagnosis

International Journal of Laboratory Hematology, EarlyView.
ABSTRACT Background Schistocytes are critical morphological markers for thrombotic microangiopathy (TMA) diagnosis. Manual identification is hampered by inconsistent standards and high interobserver variability, compromising accuracy. Although AI has advanced in hematology, AI‐enabled schistocyte classification remains under‐studied, creating a ...
Lei Shang, Chao Fang, Dongshuo Li, Xuesong Wang, Jiani Yu, Yunchu Cui, Jingliang Chen, Xuekai Liu   +7 more
wiley   +1 more source

Unsupervised Global CBC/RUO/CPD Phenotyping Identifies Haematological Clusters Enriched for Thrombocytopenia Severity and Mechanisms

International Journal of Laboratory Hematology, EarlyView.
ABSTRACT Introduction Thrombocytopenia has heterogeneous mechanisms, including peripheral destruction, consumption, and reduced marrow production. This study developed an unsupervised global complete blood count/research‐use‐only/cell population data (CBC/RUO/CPD) phenotyping model and evaluated post hoc whether thrombocytopenia severity and clinically
Mohammad A. Altememi, Emmanuel J. Favaloro, Md Zahidul Islam, Sara Ng, Danny Hsu, Abishek B. Santhakumar   +5 more
wiley   +1 more source

Severe Thrombocytopenia Associated With Glucagon‐Like‐1 Receptor Agonists (GPL‐1RA): A SONAR Report

Diabetes, Obesity and Metabolism, EarlyView.
Charles L. Bennett, Chadi Nabhan, Demir Uslu, Yogitha Dadi, David Aboulafia, Barbara Senger, Joseph Magagnoli, Kelvin Rojas, Gurdeep Singh, Linda Martin, Edward Smith, Samuel Ruder, Chien‐Hsiang Weng, Nikhil Uppal, Mia Lynch, James B. Bussel   +15 more
wiley   +1 more source

Feasibility, Safety, and Efficacy of Pulsed Field Ablation for Ventricular Arrhythmias: Insights From a Large Health System Registry in the United States

Journal of Cardiovascular Electrophysiology, EarlyView.
ABSTRACT Background Pulsed field ablation (PFA) is an emerging non‐thermal ablation modality with proven safety in atrial arrhythmia ablation. However, data on its performance for ventricular arrhythmias (VA) remain limited. Objective To evaluate the feasibility, safety, and efficacy of PFA for complex VA in a multicenter real‐world US registry ...
Narut Prasitlumkum, Juan F. Rodriguez‐Riascos, Hema Vemulapalli, Aakash Somappa, Camilo A. Estrada‐Vargas, Olubadewa A. Fatunde, Carlos A. Rojas, Poojan Prajapati, Keerthika Vijayakumar, Suraj Kapa, Duy Nguyen, Samuel Asirvatham, Malini Madhavan, Christopher McLeod, Hicham Z. El Masry, Abhishek J. Deshmukh, Danesh K. Kella, Ammar M. Killu, Siva K. Mulpuru, Mayank Sardana, Luis R. Scott, Alan M. Sugrue, Komandoor Srivathsan   +22 more
wiley   +1 more source