Results 161 to 170 of about 1,894,310 (299)

Efficacy and Safety of Subcutaneous Anifrolumab in Systemic Lupus Erythematosus: A Randomized, Phase 3 Study

open access: yesArthritis &Rheumatology, EarlyView.
Objective The multinational, phase 3, double‐blind, placebo‐controlled TULIP‐SC trial evaluated the efficacy and safety of subcutaneous anifrolumab in adults who have moderate to severe systemic lupus erythematosus (SLE) activity, despite receiving standard therapy. Methods Adults with SLE received subcutaneous anifrolumab 120 mg or placebo once weekly
Susan Manzi   +12 more
wiley   +1 more source

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

Pentoxifylline dose finding trial in preterm neonates with suspected late onset sepsis (PTX‐trial)

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Aim The aim of this study (PTX‐trial) is to determine the optimal dose of pentoxifylline (PTX) in preterm neonates (gestational age < 30 weeks) with (suspected) late onset sepsis (LONS). Methods The PTX‐trial is a prospective multicentre open‐label sequential dose‐optimization study with an adapted continual reassessment method.
Serife Kurul   +7 more
wiley   +1 more source

Prognostic impact of acute kidney injury on one-year survival in patients undergoing high-risk PCI with Impella support. [PDF]

open access: yesSci Rep
Sacha J   +53 more
europepmc   +1 more source

How the HTAR will contribute to a value‐based decision‐making for medicinal products across the EU

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
The European Union Health Technology Regulation 2021/2282 (HTAR) introduces joint assessment of health technologies (including medicinal products and medical devices) across EU Member States. It was signed into law in 2021 and came into full force in January 2025.
Roisin Adams, Michal Stanak
wiley   +1 more source

Clinically Actionable Explainable AI in Pulmonary Arterial Hypertension: Endpoints, Calibration, and External Validation. Reply to Pagnoni et al. Toward Clinically Actionable Explainable AI in Pulmonary Arterial Hypertension: Endpoints, Calibration, and External Validation. Comment on "Ledziński et al. Personalized Medicine in Pulmonary Arterial Hypertension: Utilizing Artificial Intelligence for Death Prevention. <i>J. Clin. Med.</i> 2025, <i>14</i>, 8325". [PDF]

open access: yesJ Clin Med
Ledziński Ł   +28 more
europepmc   +1 more source

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

open access: yesBritish 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   +4 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

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