Results 201 to 210 of about 285,008 (254)
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
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
Efficacy, safety and cost‐effectiveness of CAR‐T therapy
CAR T‐cells demonstrate high efficacy in blood cancers, including ALL, MM and DLBCL. Innovations target solid tumours despite challenges such as antigen escape. Combination therapies enhance the delivery and infiltration of CAR T cells. Toxicity, cost and resistance remain major barriers to clinical use.
Emina Karahmet Sher +7 more
wiley +1 more source
openaire +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
Potential for use of Al/machine learning for pharmacovigilance: Is there a role for regulators?
British Journal of Clinical Pharmacology, EarlyView.
Christina Gao +3 more
wiley +1 more source
Exploring new avenues: Psychedelic‐assisted therapy for young people
Rates of mental illness in young people are increasing, whereas the development of novel mental health treatments has not significantly progressed. Psychedelic‐assisted therapy, using substances such as psilocybin and 3,4‐methylenedioxymethamphetamine (MDMA), has shown potential in the treatment of mental illnesses in the adult population, including ...
Ioanna Artemis Vamvakopoulou +3 more
wiley +1 more source
Quality Measures Addressing Disparities to Improve Outcomes in Hand Surgery. [PDF]
Schultz EA +13 more
europepmc +1 more source
Social Determinants of Daily Step Count in Young Adults With and Without Prediabetes and Type 2 Diabetes. [PDF]
Jankowski M +6 more
europepmc +1 more source

