long term outcomes of adjuvant proton radiotherapy prt for residual pituitary adenoma pa in adults a retrospective single institute experience

Original title: “Long-term outcomes of adjuvant proton radiotherapy (PRT) for residual pituitary adenoma (PA) in adults - a retrospective, single institute experience.”

gastritis cystica profunda occurring under immunosuppression a case report of a patient with autoimmune hepatitis

Original title: “Gastritis cystica profunda occurring under immunosuppression: A case report of a patient with autoimmune hepatitis.”

type i interferon signature does not correlate with disease activity in blau syndrome

Original title: “Type I interferon signature does not correlate with disease activity in Blau syndrome.”

central nervous system multiple myeloma an update for 2026

Original title: “Central nervous system multiple myeloma: An update for 2026.”

cross sectional survey on perioperative anemia management in major abdominal surgery current practices and resource challenges among chinese surgeons

Original title: “Cross-sectional survey on perioperative anemia management in major abdominal surgery: Current practices and resource challenges among Chinese surgeons.”

Understanding the unique challenges ... for rare diseases. Exploring innovative strategies to overcome these challenges. Highlighting the importance of patient involvement and regulatory support. ... Clinical trials are the backbone of innovation in the medical field, serving as the crucial step in approving new treatments and therapies... Understanding the unique challenges in conducting clinical trials for rare diseases. Exploring innovative strategies to overcome these challenges. Highlighting the importance of patient involvement and regulatory support. ... Clinical trials are the backbone of innovation in the medical field, serving as the crucial step in approving new treatments and therapies. Despite the obstacles, researchers are employing innovative approaches to optimize rare disease clinical trials. Adaptive trial designs allow for modifications based on interim results, making studies more efficient and responsive to emerging data. Basket trials, in which a single therapeutic intervention is evaluated across multiple diseases sharing a common molecular target, also help broaden recruitment and generate robust outcomes with limited sample sizes. While the challenges of conducting clinical trials for rare diseases remain formidable, advances in technology, patient involvement, and regulatory incentives are steadily breaking down barriers. Collaboration among sponsors, researchers, advocacy groups, and regulators is critical to maintaining momentum and ensuring that the needs of rare disease patients are not overlooked. As awareness grows and strategies continue to evolve, the path forward promises more hope, improved therapies, and, ultimately, better lives for millions affected by rare diseases worldwide. Additionally, the lack of historical clinical data for rare diseases can hinder the development of appropriate trial methodologies. Ethical considerations, such as the feasibility of using placebos in conditions where no treatment is available, further complicate the research process.

Original title: “Overcoming Key Challenges in Conducting Clinical Trials”

discovery and optimization of marstacimab a human monoclonal antibody targeting tissue factor pathway inhibitor for the treatment of hemophilia a and b

Original title: “Discovery and optimization of marstacimab, a human monoclonal antibody targeting tissue factor pathway inhibitor for the treatment of hemophilia A and B.”

circulating igf2bp3 enables risk stratification and predicts treatment response in ewing sarcoma

Original title: “Circulating IGF2BP3 enables risk stratification and predicts treatment response in Ewing sarcoma.”

risk factors associated with apyrexia during bacteraemia a prospective multicenter cohort study

Original title: “Risk factors associated with apyrexia during bacteraemia: a prospective multicenter cohort study.”

transformer based deep learning algorithm for diagnosing spinal infections on axial non contrast computed tomography images a dual center retrospective study

Original title: “A transformer-based deep learning algorithm for diagnosing spinal infections on axial non-contrast computed tomography images: a dual-center retrospective study.”

anaphylaxis during hemodialysis associated with a polysulfone membrane evidence from basophil activation testing

Original title: “Anaphylaxis during hemodialysis associated with a polysulfone membrane: evidence from basophil activation testing.”

unveiling the variations in penile cancer care identifying knowledge gaps and research priorities

Original title: “Unveiling the variations in penile cancer care: identifying knowledge gaps and research priorities.”

peripheral t cell lymphoma in japan real world patient characteristics treatment patterns healthcare resource utilization and costs

Original title: “Peripheral T-cell Lymphoma in Japan: Real-World Patient Characteristics, Treatment Patterns, Healthcare Resource Utilization, and Costs.”

epidemiology histopathologic patterns and clinical characteristics of gestational trophoblastic diseases in ethiopia a systematic review and meta analysis study

Original title: “Epidemiology, Histopathologic Patterns, and Clinical Characteristics of Gestational Trophoblastic Diseases in Ethiopia: A Systematic Review and Meta-Analysis Study.”

evaluating the impact of different natural history modeling methods on cost effectiveness decisions a case study in duchenne muscular dystrophy

Original title: “Evaluating the Impact of Different Natural History Modeling Methods on Cost-Effectiveness Decisions: A Case Study in Duchenne Muscular Dystrophy.”

acute and chronic kidney injury following covid 19 infection and vaccination a narrative review

Original title: “Acute and chronic kidney injury following COVID-19 infection and vaccination: a narrative review.”

real world treatment and outcomes in hairy cell leukemia a multicenter retrospective cohort including braf inhibitor therapy

Original title: “Real-world treatment and outcomes in hairy cell leukemia: a multicenter retrospective cohort including BRAF inhibitor therapy.”

genetically supported drug target prioritization for rare diseases

Original title: “Genetically supported drug target prioritization for rare diseases.”