British 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
Macroeconomic-aware forecasting of construction costs in developing countries: Using gated recurrent unit and long short-term memory deep learning framework. [PDF]
PLoS OneAlzara M +6 more
europepmc +1 more source
Facilitators and barriers to adherence to severe malaria treatment guidelines in children at Mbale Regional Referral Hospital, Uganda: a health workers' perspective. [PDF]
Malar JChelangat SC +11 more
europepmc +1 more source
How the HTAR will contribute to a value‐based decision‐making for medicinal products across the EU
British 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
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Children's perspectives of their everyday food practices: insights to inform policy and interventions. [PDF]
PLoS OneWright-Pedersen S, Vidgen H, Gallegos D.
europepmc +1 more source
British Journal of Clinical Pharmacology, EarlyView.Abstract Despite regulatory progress being made in the past two decades, off‐label drug use in paediatrics remains pervasive, with prevalence estimated between 3% and 97% of prescriptions across different clinical settings. Off‐label use—defined as prescribing outside the conditions described in the Summary of Product Characteristics (SmPC)—is often ...
Tjitske M. van der Zanden +3 more
wiley +1 more source
Stakeholder Perspectives on Applying for the Fresh Fruit and Vegetable Program in Low-Resource Schools. [PDF]
J Sch HealthHoy K +4 more
europepmc +1 more source
Zuranolone: A case study in (regulatory) rush to judgement?
British Journal of Clinical Pharmacology, EarlyView.Abstract Sage, in collaboration with Biogen, submitted a new drug approval for zuranolone for postpartum depression (PPD) and major depressive disorder (MDD) in December 2022. In August 2023, the US Food and Drug Administration granted approval for PPD but denied approval for MDD.
Lisa Cosgrove +4 more
wiley +1 more source
DeFiTrustChain: A DeFi-Enabled NFT and Escrow Framework for Secure Automotive Supply Chains in Smart Cities. [PDF]
Sensors (Basel)Kurde A, Singh SK, Alotaibi A.
europepmc +1 more source
British Journal of Clinical Pharmacology, EarlyView.Abstract Aim To identify safety concerns reported by coroners following fentanyl patch‐related deaths in England, Wales and Northern Ireland, and determine differences in coronial reporting. Methods A systematic case series linking the National Programme on Substance Use Mortality (NPSUM) and the Preventable Deaths Tracker (PDT) (https ...
Eman Mshari +2 more
wiley +1 more source