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
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
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
Use of pressure muscle index to guide pressure support ventilation setting: a study protocol and statistical plan for a prospective randomised controlled proof-of-concept trial. [PDF]
Gao R +4 more
europepmc +1 more source
Aim This report presents a series of 16 patients who were admitted to the emergency department following confirmed intake of the potent synthetic cannabinoid ADB‐CHMINACA. Methods The cases are drawn from a prospective observational study following the recreational use of synthetic cannabinoids.
Maren Hermanns‐Clausen +7 more
wiley +1 more source
The effects of real-time waveform analysis software on patient ventilator synchronization during pressure support ventilation: a randomized crossover physiological study. [PDF]
Nakornnoi B, Tscheikuna J, Rittayamai N.
europepmc +1 more source
Abstract Aim Mosliciguat (BAY 1237592), the first soluble guanylate cyclase (sGC) activator in a dry powder inhaler (DPI), could potentially treat pulmonary hypertension with a more targeted delivery than systemic sGC stimulators. We report safety and tolerability of mosliciguat in healthy men.
Johannes Nagelschmitz +7 more
wiley +1 more source
Development of a nalmefene formulation suitable for a community‐use auto‐injector
Aim Time to onset of reversal is critical when treating opioid‐induced respiratory depression and hypoxia. This study aimed to develop a formulation of the opioid antagonist nalmefene with an increased absorption rate and reduced time to onset of reversal following intramuscular administration. Methods Several absorption enhancers were screened in rats,
Alessandra Cipriano +6 more
wiley +1 more source
The level of partial pressure of carbon dioxide affects organ perfusion in respiratory failure patients undergoing pressure support ventilation with venovenous extracorporeal membrane oxygenation: a prospective study. [PDF]
Zhou Y +7 more
europepmc +1 more source
ABSTRACT The biopharmaceutical industry increasingly relies on single‐use technologies (SUT) for operational flexibility. For a long time, SUT was considered more sustainable than stainless‐steel (SST) systems due to water and energy savings. This study re‐evaluates this paradigm via a detailed bottom‐up analysis of the SUT carbon footprint at the 2000
Jan Reiners +4 more
wiley +1 more source

