The Impact of Clinical Trial Participation on Quality of Life in Duchenne Muscular Dystrophy [0.03%]
杜氏肌营养不良症临床试验参与对生活质量的影响
Aidan W Leffler,Graham J Leffler,Marielle G Contesse
Aidan W Leffler
Background and objectives: While there has been an increase in Duchenne muscular dystrophy (DMD) clinical trials over the last couple of decades, there is still no cure. Many individuals with DMD spend much of their lives...
Animal Models for Drug Discovery in Schizophrenia: A Critical View from a Clinical Perspective [0.03%]
从临床角度对精神分裂症药物研发动物模型的批判性观点
Inga Dammann-Bawadkji,F Markus Leweke,Cathrin Rohleder
Inga Dammann-Bawadkji
Schizophrenia is a complex neuropsychiatric disorder manifesting with diverse positive and negative symptoms as well as cognitive impairments. Current antipsychotics primarily address positive symptoms and frequently cause substantial side ...
Amit Lalwani,Jon Zdon,Maaike Addicks et al.
Amit Lalwani et al.
Digital transformation is increasingly shaping how the Medical Affairs (MA) function generates insights, engages stakeholders, and demonstrates value within the pharmaceutical industry. This article examines how MA professionals can navigat...
Medical Affairs in the Age of Complexity: From Scientific Support to Strategic Enterprise Integrator [0.03%]
复杂时代下的医学事务：从科学支持到战略性企业整合者
Mussa Arvani
Mussa Arvani
Regulatory acceleration, expanded reliance on real-world evidence (RWE), and increasing integration of artificial intelligence (AI) into healthcare systems have substantially reshaped expectations placed on Medical Affairs. Accelerated and ...
Regulatory Considerations for Combined Drug-Device Studies in Europe: A Case Study Involving Bile Sampling [0.03%]
欧洲联合药械临床试验的监管考量：一项胆汁采样案例研究
Hans-Peter Podhaisky,Emiko Tsuruta,Isabel Piel et al.
Hans-Peter Podhaisky et al.
Language as an Eligibility Criterion in Pediatric Study Protocols Conducted in Europe (2007-2024) Reported in the ClinicalTrials.gov Database [0.03%]
欧洲（2007-2024）儿科研究方案中的语言入选标准：来自ClinicalTrials.gov数据库的报道
Begonya Nafria,Alexander Perera,Segolene Gaillard et al.
Begonya Nafria et al.
Background: One of the challenges in accessing cross-border clinical trials involving international participants in Europe is language diversity, with 32 official languages in the European continent. Some patients have re...
A Framework for Standardising Toxicity Management Guidelines in Oncology Clinical Trials [0.03%]
肿瘤学临床试验中毒性管理指南标准化框架
Sanjana Singarayer,Heidi Lloyd-Williams,Abigail Morrison et al.
Sanjana Singarayer et al.
For new investigational medicinal products (IMPs) entering clinical development, toxicity management guidelines (TMGs) can be developed as an addition to the clinical study protocol. These guidelines inform best clinical practice for the tr...
Correction: Paediatric Phase I/II Clinical Trial Assessments Under the New EU Clinical Trial Regulation: A Descriptive Analysis [0.03%]
关于欧盟临床试验新法规下儿童一期和二期临床试验评估的更正：描述性分析
Laura Fankhauser,Franca Ligas,Giovanni Lesa et al.
Laura Fankhauser et al.
Published Erratum
Pharmaceutical medicine. 2026 Jun 19. DOI:10.1007/s40290-026-00616-y 2026
An Expert Consensus-Based Review of Considerations for Developing a Quality Management System Framework for Studies Utilising Secondary Data for Real-World Evidence Generation [0.03%]
基于专家共识的二次利用数据开展真实世界证据研究的质量管理体系框架考量评审报告
Deborah Layton,Antonella Porta,Dony Patel et al.
Deborah Layton et al.
High-quality pharmacoepidemiological research is essential for credible real-world evidence (RWE). This expert consensus-based review explores how pharmaceutical Quality Management Systems (QMS) developed for studies using primary data coll...
Optimising Formulations for Paediatric Patients with Inherited Metabolic Disorders: The Case Study of Levocarnitine [0.03%]
遗传性代谢紊乱患儿给药方案的优化——左卡尼丁治疗的案例研究
Stephen Tomlin,Chris Mühlhausen,Andriy Krendyukov et al.
Stephen Tomlin et al.
Inherited metabolic disorders (IMDs) are rare genetic conditions that disrupt normal biochemical pathways, leading to potentially life-threatening metabolic imbalances. Early diagnosis, often through newborn screening, and treatment can sig...