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
Epidemiology of Incidence and Mortality Due to Head and Neck Cancer in Poland in 2000 to 2022. [PDF]
Zaborska P +2 more
europepmc +1 more source
Pentoxifylline dose finding trial in preterm neonates with suspected late onset sepsis (PTX‐trial)
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
How the HTAR will contribute to a value‐based decision‐making for medicinal products across the EU
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
Correction: Multidimensional assessment of urinary dysfunction and quality of life after cervical cancer treatment: a multicenter study integrating urodynamics and patient-reported outcomes. [PDF]
Bereza K +5 more
europepmc +1 more source
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
Nationwide Characteristics of Patients Admitted to Hospital for the First Time Due to Primary Liver Cancer and Intrahepatic Bile Duct Cancer in Poland in 2012-2023. [PDF]
Genowska A +9 more
europepmc +1 more source
Abstract Aim Tezepelumab is a human monoclonal antibody that blocks the activity of thymic stromal lymphopoietin and is approved for adolescents and adults with severe asthma. This analysis assessed pharmacokinetics (PK), pharmacodynamics and safety of tezepelumab up to 85 days postdose in adolescents and children.
Anna Lundahl +14 more
wiley +1 more source
OnabotulinumtoxinA for chronic migraine: A real-life multicenter study of 479 patients. [PDF]
Waliszewska-Prosół M +25 more
europepmc +1 more source
Abstract Aim Balcinrenone (previously AZD9977) is a novel selective non‐steroidal mineralocorticoid receptor antagonist (MRA) with a differential mechanism of action relative to approved MRAs. The aim of this trial was to assess the pharmacokinetics, safety and tolerability of balcinrenone in participants with mild and moderate hepatic impairment ...
Anna L. Eriksson +8 more
wiley +1 more source

