Results 61 to 70 of about 86,084 (265)

Assessment of joint health in patients receiving prophylaxis for haemophilia A: a cross-sectional survey in five European countries

BMJ Open
Objectives To evaluate joint health, pain and health-related quality of life (HRQoL) in patients with moderate/severe haemophilia A in Europe.Design Multinational, cross-sectional survey, with retrospective data collection.
Zalmai Hakimi, Hortensia De la Corte-Rodríguez, Linda Bystrická, Nathan Ball, Stevie Olsen, Keisha Golden, Nana Kragh   +6 more
doaj   +1 more source

Perec orphelin : d’un père, d’un peuple, d’un e

Recherches & Travaux, 2020
Deeply shaped by the apparently contradictory themes of lack and excess, Georges Perec’s work perpetually and mechanically reactivates the topoi of absence and searches for an overflow which is itself just another figure of loss.
Benoît Abert
doaj   +1 more source

Do drugs approved via expedited approval pathways have therapeutic advantages? A systematic review and meta‐analysis

British Journal of Clinical Pharmacology, EarlyView.
Regulators use expedited approval pathways to speed market approval and patient access to promising new drugs. However, there is uncertainty about whether these pathways are successful in approving drugs with significant therapeutic advantages. This systematic review aims to examine the safety, effectiveness and cost‐effectiveness of drugs approved via
Ashleigh Hooimeyer, Eliza J. McEwin, Zhicheng Wang, Joel Lexchin, Barbara Mintzes   +4 more
wiley   +1 more source

Decoding RNA regulation: Challenges and opportunities for RNA‐based therapies in Europe

British Journal of Clinical Pharmacology, EarlyView.
Abstract RNA‐based medicinal products represent a promising frontier in personalised medicine, offering sequence‐specific disease targeting at various molecular levels, yet their clinical translation in the European Union (EU) may be hindered by regulatory uncertainty around definitions and evidence requirements; this study therefore aims to identify ...
Olivia C. Lewis, Christine Leopold, Joyce M. Hoek, Raymond M. Schiffelers, Marie L. De Bruin   +4 more
wiley   +1 more source

The Kelch-Repeat Superfamily Gene SiNL4 Regulates the Leaf Width in Foxtail Millet

Plants
The Kelch-repeat superfamily genes played important roles in regulating plant growth and development; however, their functions in foxtail millet (Setaria italica) have not yet been characterized.
Yuqin Zhao, Yixuan Ma, Yanyu Yang, Lejie Yang, Lu Chen, Tianguo Wang, Shiyuan Wang, Kai Zhao, Xiaorui Li, Shuqi Dong, Hongzhi Wang, Xiaoqian Chu, Jiagang Wang, Lulu Gao, Guanghui Yang   +14 more
doaj   +1 more source

Advanced therapy medicinal products (ATMPs) – An analysis of the global regulatory status

British Journal of Clinical Pharmacology, EarlyView.
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, Freja Karuna Hemmingsen Sørup, Marie Lund, Tonny Studsgaard Petersen, Kristian Karstoft   +4 more
wiley   +1 more source

Long standing eculizumab treatment without anticoagulant therapy in high-risk thrombogenic paroxysmal nocturnal hemoglobinuria

Hematology Reports, 2015
Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-orphan disease affecting all hematopoietic cell types. The abnormality of red blood cells in this disease predisposes to intravascular complement-mediated hemolysis.
Hassan A. Al-Jafar, Salma M. AlDallal, Haifa A. Askar, Ali M. Aljeraiwi, Ahmad Al-Alansari   +4 more
doaj   +1 more source

Infusion rate adjustment in enzyme replacement therapy with pabinafusp alfa for mucopolysaccharidosis II

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aims Enzyme replacement therapy (ERT) for mucopolysaccharidosis II (MPS II) requires long‐term, weekly intravenous infusions often lasting over 3 h each time, which can burden paediatric patients and caregivers and negatively affect their quality of life and treatment compliance.
Kimitoshi Nakamura, Norio Sakai, Hideaki Hirai, Naoko Takasao, Ryo Ibaraki, Tatsuyoshi Yamamoto, Yuji Sato   +6 more
wiley   +1 more source

Impact of CFTR Modulators on Longitudinal Cystic Fibrosis Survival and Mortality: Review and Secondary Analysis

Pulmonary Therapy
Introduction Cystic fibrosis (CF) transmembrane conductance regulator modulators (CFTRm) have transformed CF care, shifting treatment from only managing symptoms to also addressing the underlying defects that cause CF.
Jaime L. Rubin, Craig McKinnon, Gabriel Ghizzi Pedra, Devon A. Morgan, Kimberly Zweig, Theodore G. Liou   +5 more
doaj   +1 more source

Effects of Institutionalization and Parental Living Status on Children’s Self-Esteem, and Externalizing and Internalizing Problems in Rwanda

Frontiers in Psychiatry, 2019
The negative effects of institutionalization on children’s wellbeing and psychological adjustment have been extensively documented. Throughout the world, particularly in developing countries, many children in residential child care institutions known as ...
Epaphrodite Nsabimana, Epaphrodite Nsabimana, Eugène Rutembesa, Peter Wilhelm, Chantal Martin-Soelch   +4 more
doaj   +1 more source