Results 81 to 90 of about 43,399 (123)

Health Care, Technological Change, and Altruistic Consumption Externalities [PDF]

Traditional economic analysis has proposed well known remedies to deal with consumption externalities and inefficient technological change in isolation, but lacks a general framework for addressing them jointly. We argue that the joint determination of R&
Anupam Jena, Stephane Mechoulan, Tomas Philipson   +2 more
core  

Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Haemoglobinuria

European Journal of Haematology, EarlyView.
ABSTRACT Background Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra‐rare, acquired non‐malignant haematological disorder characterised by thrombosis risk, serious complications and debilitating symptoms in untreated patients. Objective This anchored indirect treatment comparison (ITC) evaluated efficacy data between proximal complement 3 ...
Regis Peffault de Latour, Austin Kulasekararaj, David Dingli, Koo Wilson, Piotr Wojciechowski, Zalmai Hakimi, Jameel Nazir, Barbara Czech, Peter Hillmen, Jeff Szer   +9 more
wiley   +1 more source

Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study

European Journal of Haematology, EarlyView.
ABSTRACT Objective To report data from an ITI chart review study (NCT03951103) for first‐time and rescue ITI with recombinant factor VIII Fc fusion protein (rFVIIIFc) in persons with haemophilia A. Methods Retrospective and prospective real‐world data are reported from a non‐interventional, multicentre study of patients who had been or were currently ...
Robert Klamroth, Mahasen Al Saleh, Heidi Glosli, Michele Schiavulli, Benoît Guillet, Linda Bystrická, Anton Schönstein, Stefan Lethagen   +7 more
wiley   +1 more source

Making rareness count: testing and pricing orphan drugs [PDF]

, 2016
This paper examines the testing and the pricing of orphan drugs, e.g. drugs for patients suffering from rare diseases. Due to the small size of these populations, orphan molecules question established evidentiary practices, namely randomized controlled ...
Doganova, Liliana, Rabeharisoa, Vololona
core   +3 more sources

Comorbidity and Mortality in Men and Women With Haemophilia in Three Nordic Countries–Comparisons to Matched Controls

Haemophilia, EarlyView.
ABSTRACT Introduction Comorbidities and public health conditions in haemophilia are receiving increasing attention. Aim To analyse the prevalence of comorbidities and mortality in people with haemophilia (PwH) compared to matched controls in subgroups (factor consumption and sex).
Katarina Steen Carlsson, Jan Astermark, Fariba Baghaei, Elisabeth Brodin, Eva Funding, Margareta Holmström, Klaus Österholm, Sofia Bergenstråle, Stefan Lethagen   +8 more
wiley   +1 more source

Profiling of Anti‐FVIII Antibodies in Acquired Haemophilia A: ‘Insights into Domain Specificity, Isotype Variability, and Clinical Correlations’

Haemophilia, EarlyView.
ABSTRACT Introduction Acquired haemophilia A (AHA) is a rare autoimmune disorder caused by autoantibodies against coagulation factor VIII (FVIII), resulting in significant bleeding risks. Aim To characterize the anti‐FVIII antibody profile in AHA patients by assessing isotypes, subclasses, and correlations with key clinical parameters.
Ann‐Cristin Berkemeier, Isabell Matuschek, Katrin Hartlieb, Thilo Albert, Natascha Marquardt, Johannes Oldenburg, Behnaz Pezeshkpoor   +6 more
wiley   +1 more source

ROS1 immunohistochemistry as a potential predictive biomarker for ROS1‐targeted therapy in breast cancer: impact of antibody clone selection

Histopathology, EarlyView.
ROS1 antibody clones are not interchangeable in the breast cancer setting and highlight major discordances in ROS1 status according to antibody clone utilised. Aims Invasive lobular carcinoma (ILC) may show targetable vulnerabilities secondary to the characteristic loss of the cell adhesion protein E‐cadherin.
Anna Sokolova, Vaibhavi Joshi, Haarika Chittoory, Michael Walsh, Malcolm Lim, Jamie R Kutasovic, Kaltin Ferguson, Peter T Simpson, Sunil R Lakhani, Amy E McCart Reed   +9 more
wiley   +1 more source

Are single nucleotide polymorphisms underutilized for guiding treatment of inflammatory bowel disease?

Immunology &Cell Biology, EarlyView.
Patients with inflammatory bowel disease (IBD) show variation in their response to therapeutics. This review explores the contrast between heritable single nucleotide polymorphisms (SNPs) that contribute the pathogenesis of IBD, versus those that associate with response to treatments.
Jildou van der Werf, Nicholas Ian Fleming   +1 more
wiley   +1 more source

Narcolepsy and rapid eye movement sleep

Journal of Sleep Research, Volume 34, Issue 2, April 2025.
Summary Since the first description of narcolepsy at the end of the 19th Century, great progress has been made. The disease is nowadays distinguished as narcolepsy type 1 and type 2. In the 1960s, the discovery of rapid eye movement sleep at sleep onset led to improved understanding of core sleep‐related disease symptoms of the disease (excessive ...
Francesco Biscarini, Lucie Barateau, Fabio Pizza, Giuseppe Plazzi, Yves Dauvilliers   +4 more
wiley   +1 more source

Actigraphy against 32‐hour polysomnography in patients with suspected idiopathic hypersomnia

Journal of Sleep Research, EarlyView.
Summary Actigraphy, a tool known for investigating sleep–wake patterns at home, lacks scientific validation in hypersomnolent subjects. We aim to validate an actigraphy‐based sleep–wake prediction algorithm against 32‐h continuous polysomnography in patients with suspected idiopathic hypersomnia, and to compare its performance to predict sleep–wake ...
Tugdual Adam, Jérôme Tanty, Lucie Barateau, Yves Dauvilliers   +3 more
wiley   +1 more source