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Incidence, case fatality, and functional outcome of intracerebral haemorrhage, according to age, sex, and country income level: a systematic review and meta-analysis.
Lancet Reg Health Eur
February 2025
Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Centre, Geert Grooteplein Zuid 10, 6525 GA, Nijmegen, the Netherlands.
Background: Intracerebral haemorrhage (ICH) accounts for approximately 28% of all strokes worldwide. ICH has a high case fatality, and only few survivors recover to independent living. Over the past decades, demographic changes, and changes in prevalence and management of risk factors may have influenced incidence.
View Article and Find Full Text PDFOptimizing rare disorder trials: a phase 1a/1b randomized study of KL1333 in adults with mitochondrial disease.
Brain
January 2025
Department of Neuromuscular Diseases, University College London Queen Square Institute of Neurology, London WC1N 3BG, UK.
Over the past two decades there has been increased interest in orphan drug development for rare diseases. However, hurdles to clinical trial design for these disorders remain. This phase 1a/1b study addressed several challenges, while evaluating the safety and tolerability of the novel oral molecule KL1333 in healthy volunteers and subjects with primary mitochondrial disease.
View Article and Find Full Text PDFPegylated interferon: the who, why, and how.
Hematology Am Soc Hematol Educ Program
December 2024
Hopital Saint-Louis, Paris Cité University, Inserm CIC 1427, Paris, France.
Interferon alpha (IFN-α) is a fascinating molecule with many biological properties yet to be fully understood. Among these properties, several have demonstrated usefulness for targeting malignant cells, including hematopoietic cells from patients with myeloproliferative neoplasms. Indeed, IFN-α has been used for decades across all myeloproliferative neoplasms, but only recently a new form, ropegIFN-α2b, was approved to treat patients with polycythemia vera.
View Article and Find Full Text PDFTherapeutic orphans, off-label, pediatric drug development: towards reasonable pharmacotherapy for minors.
Expert Opin Pharmacother
December 2024
Pediatric Neurology and Muscular Diseases Unit, Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, Giannina Gaslini Institute, University of Genova, Genova, Italy.
Introduction: The concept that children are therapeutic orphans emerged in the 1960s, triggering eventually worldwide legislation to facilitate pediatric studies, called 'Pediatric Drug Development (PDD).' However, PDD's true aim is not better medicines for children but labels in minors; minors are not another species.
Areas Covered: Absorption, distribution, metabolism, and excretion (ADME) differ in preterm newborns, but babies mature.
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