acute intestinal ischemia secondary to splanchnic venous thrombosis caused by a novel pathogenic pros1 mutation a case report

Original title: “Acute intestinal ischemia secondary to splanchnic venous thrombosis caused by a novel pathogenic PROS1 mutation: a case report.”

sod1 amyotrophic lateral sclerosis associated with neurosarcoidosis a case report and review of the literature

Original title: “SOD1 amyotrophic lateral sclerosis associated with Neurosarcoidosis: a case report and review of the literature.”

fatal pediatric case of febrile ulceronecrotic mucha habermann disease diagnostic and therapeutic challenges a case report

Original title: “A fatal pediatric case of febrile ulceronecrotic Mucha-Habermann disease: diagnostic and therapeutic challenges: a case report.”

nosocomial outbreaks with rare yeasts trends characteristics and preventive measures

Original title: “Nosocomial outbreaks with rare yeasts: trends, characteristics and preventive measures.”

when life gives you limes it gives you candida a case of lime contaminated iv injection

Original title: “When life gives you limes… it gives you Candida: a case of lime-contaminated IV injection.”

traumatic intraperitoneal rupture of a hepatic hydatid cyst presenting as refractory anaphylactic shock a rare case report

Original title: “Traumatic intraperitoneal rupture of a hepatic hydatid cyst presenting as refractory anaphylactic shock: a rare case report.”

optimizing rare neurological disease trials bayesian frameworks and hierarchical models for improved efficiency in clinical trial design

Original title: “Optimizing rare neurological disease trials: Bayesian frameworks and hierarchical models for improved efficiency in clinical trial design.”

Early integration of clinical, operational, and patient-level insights during trial development can help close the gap between evidence generation and commercial performance, improving alignment between product value and real-world adoption. This evaluation starts during the recruitment phase of a clinical trial and may serve as an area to direct strategies toward during the marketing phase of contacting HCPs to present the drug. Such assessment is essentially implemented in rare disease studies, which in many cases show gaps in diagnosis and may delay the start of patients’ treatment. This encompasses awareness of the disease to be treated and the patient journey, as well as understanding the new mechanisms of actions to treat the target disease. It is important to understand whether the medical field is prepared to receive a new technology to be used for a specific purpose. This helps the internal medical affairs (MAs) team and commercial to discuss and influence HCPs on the findings of a new drug and how it may improve the existing treatments. Such educational support can be identified in the beginning of clinical trials based on doubts and questions from investigators and site staff in investigators’ meetings and initiation visits, as well as throughout the study during recruitment period. As a second domain, insights into drug handling and operational feasibility during clinical trials can provide early signals regarding drug administration complexity, storage and logistics requirements, and site workflow integration. Operational inefficiencies often predict real-world adoption barriers. Evidence suggests that therapies requiring complex administration face slower uptake unless supported by robust infrastructure and training.5,7 Moreover, logistical hurdles may impact drug delivery in remote healthcare institutions where deficiencies in transportation lead to a risk in the product integrity. Clinical trials generate a rich ecosystem of insights beyond primary endpoints; thus, four key domains of translational values can be considered: Investigator’s experience and perception of the drug. Operational feasibility and drug handling constraints. Patient heterogeneity-profile, adherence, and treatment patterns.

Original title: “Translating Clinical Trial Insights into Commercial Strategy: Leveraging Study Data Across the Product Lifecycle | Applied Clinical Trials Online”

direct oral anticoagulant consumption and the risk of acute interstitial lung diseases a case crossover study using japanese administrative data

Original title: “Direct Oral Anticoagulant Consumption and the Risk of Acute Interstitial Lung Diseases: A Case-Crossover Study Using Japanese Administrative Data.”

pontocerebellar hypoplasia linked mutations of the deadenylase target of egr1 toe1 impair thermal stability ribonuclease activity and oligomerization

Original title: “Pontocerebellar Hypoplasia linked mutations of the deadenylase Target of EGR1 (TOE1) impair thermal stability, ribonuclease activity, and oligomerization.”

multifunctional spatiotemporally programmed microneedle patches for the reconstruction of antibacterial and immunoregenerative homeostasis in periodontitis therapy

Original title: “Multifunctional spatiotemporally programmed microneedle patches for the reconstruction of antibacterial and immunoregenerative homeostasis in periodontitis therapy.”

strengthening the evidence base for precision medicine through the utilization of real world data and real world evidence a narrative review from the u s perspective

Original title: “Strengthening the evidence base for precision medicine through the utilization of real-world data and real-world evidence: a narrative review from the U.S. perspective.”

orbital apex syndrome secondary to a dental implant infection caused by staphylococcus lugdunensis

Original title: “Orbital Apex Syndrome Secondary to a Dental Implant Infection Caused by Staphylococcus lugdunensis.”

case of bilateral but not unilateral insufficient calcaneus fractures in a patient with rheumatoid arthritis suffering from talalgia

Original title: “A case of bilateral but not unilateral insufficient calcaneus fractures in a patient with rheumatoid arthritis suffering from talalgia.”

coexistence of parkinson s disease and myasthenia gravis with symptom exacerbation induced by biperiden a case report

Original title: “Coexistence of Parkinson's Disease and Myasthenia Gravis with Symptom Exacerbation Induced by Biperiden: A Case Report.”

severe hdl c deficiency caused by acquired lcat deficiency a case report with lipidomic profiling and anti lcat autoantibody detection

Original title: “Severe HDL-C Deficiency Caused by Acquired LCAT Deficiency: A Case Report with Lipidomic Profiling and Anti-LCAT Autoantibody Detection.”

deep medullary vein drainage and unfavorable outcomes in stroke patients after endovascular therapy a retrospective study

Original title: “Deep medullary vein drainage and unfavorable outcomes in stroke patients after endovascular therapy: a retrospective study.”

biatrial cardiac extension of burkitt lymphoma a case report of successful surgical correction

Original title: “BiatriaL cardiac extension of Burkitt lymphoma: a case report of successful surgical correction.”

clinically isolated joint involvement is a distinct entity in whipple s disease

Original title: “Clinically isolated joint involvement is a distinct entity in Whipple's disease.”

development and validation of a nomogram for early prognostic prediction in childhood aplastic anemia receiving cyclosporine monotherapy

Original title: “Development and validation of a nomogram for early prognostic prediction in childhood aplastic anemia receiving cyclosporine monotherapy.”