Results 221 to 230 of about 349,027 (301)

Switching disease‐modifying therapies in patients with spinal muscular atrophy: A systematic review on effectiveness outcomes

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
With multiple disease‐modifying therapies now available, treatment switching has become an important clinical consideration in the management of spinal muscular atrophy (SMA). While some switches are prompted by suboptimal clinical response, more commonly they are driven by treatment burden, convenience, or adverse events.
Andrej Belančić   +4 more
wiley   +1 more source

Cost‐utility analysis of nusinersen–risdiplam switch in patients with spinal muscular atrophy in Croatia: A discrete event simulation model

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić   +4 more
wiley   +1 more source

Paediatric development of radiopharmaceutical imaging agents and radioligand therapeutics

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Abstract This review focuses on the development of radiopharmaceutical imaging agents and radioligand therapeutics for paediatric use. Nuclear medicine plays an important role in the diagnosis and treatment of various childhood conditions, including cancers, infections and brain disorders.
Justin L. Hay   +5 more
wiley   +1 more source

Successful Versus Failed Transition From Controlled Ventilation to Pressure Support Ventilation in COVID-19 Patients: A Retrospective Cohort Study. [PDF]

open access: yesCrit Care Explor
Polo Friz M   +27 more
europepmc   +1 more source

Intravenous lanadelumab for the treatment of moderately ill COVID‐19 patients

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Aims Kallikrein‐kinin system (KKS) dysregulation is hypothesized to play a pathogenetic role in COVID‐19‐associated pulmonary oedema. To investigate the efficacy and safety of intravenous lanadelumab, a monoclonal antibody that inhibits plasma kallikrein, in COVID‐19, we conducted a phase 2, open‐label, randomized‐controlled, proof‐of‐concept ...
Job J. Engel   +12 more
wiley   +1 more source

The evolving therapeutic landscape of spinal muscular atrophy – A scoping review of investigational agents, emerging delivery technologies and strategic innovations

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić   +7 more
wiley   +1 more source

Understanding the paradox of primary teacher shortage in low‐income countries: Insights from Malawi

open access: yesBritish Educational Research Journal, EarlyView.
Abstract Malawi faces a persistent and chronic teacher shortage, particularly at the primary school level, with much of the challenge attributed to teacher supply constraints. While the causes of teacher shortages are known to be complex and context‐dependent, there remains a lack of in‐depth, country‐specific research to guide effective policies ...
Peter Mtika, Edward M. Sosu
wiley   +1 more source

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