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
Use of indocyanine green in thoracic surgery: a literature review. [PDF]
Walczak K +5 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
Multidisciplinary management of acute type A aortic dissection in a pregnant woman with Marfan syndrome. [PDF]
Jodłowski M +6 more
europepmc +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
Contralateral thoracic duct injury after unilateral cervical stab wound: a rare cause of chylothorax. [PDF]
Çetinkaya Ç.
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
Rare disease, common struggles: quality of life, caregiver burden and financial wellbeing of family caregivers in Poland. [PDF]
Domaradzki J, Jabkowski P, Walkowiak D.
europepmc +1 more source
‘Where are the adults?’: Troubling child‐activism and children's political participation
Abstract Children's political participation is a well‐established theme in childhood studies. In this article we offer an original account of child activism that takes into account the entangled and emergent aspect of children as activists. We begin with a historical and a conceptual review, noting the importance of mid‐20th century developments such ...
Sharon Hunter, Claire Cassidy
wiley +1 more source
Minimally invasive approach for ascending aorta aneurysm replacement - next steps in minimally invasive cardiac surgery: a case series. [PDF]
Janeczek M +3 more
europepmc +1 more source

