from the archives of md anderson cancer center crystal storing histiocytosis associated with b cell lymphoma

Original title: “From the archives of MD Anderson Cancer Center: Crystal-storing histiocytosis associated with B-cell lymphoma.”

primary malignant pericardial epithelioid mesothelioma presenting as cardiac tamponade a case report with multimodality imaging and radiologic pathologic correlation

Original title: “Primary Malignant Pericardial Epithelioid Mesothelioma Presenting as Cardiac Tamponade: A Case Report With Multimodality Imaging and Radiologic-Pathologic Correlation.”

prolonged neonatal phosphate retention and transient hypercalcemia following antenatal burosumab exposure a pharmacovigilance alert

Original title: “Prolonged neonatal phosphate retention and transient hypercalcemia following antenatal Burosumab exposure: a pharmacovigilance alert.”

perianal crohn disease in a patient with congenital afibrinogenemia a multidisciplinary therapeutic management challenge

Original title: “Perianal Crohn Disease in a Patient With Congenital Afibrinogenemia: A Multidisciplinary Therapeutic Management Challenge.”

interstitial lung disease in mixed connective tissue disease insights from an electronic health record based retrospective cohort study

Original title: “Interstitial lung disease in mixed connective tissue disease: Insights from an electronic health record-based retrospective cohort study.”

restorative challenges reconstruction of large axillary defects following surgery for extramammary paget s disease

Original title: “Restorative Challenges: Reconstruction of Large Axillary Defects Following Surgery for Extramammary Paget's Disease.”

purple urine bag syndrome in a 26 year old woman living with hiv and metastatic spinal cord compression a case report

Original title: “Purple Urine Bag Syndrome in a 26-Year-Old Woman Living with HIV and Metastatic Spinal Cord Compression: A Case Report.”

GSK claimed the result was a first for an ADC aimed at the “B7-H3” protein, an increasingly popular target of late among cancer drugmakers.

Original title: “GSK, Hansoh ADC extends survival in lung cancer study”

The lineup at the Alzheimer’s Association International Conference will provide critical insight into where the industry is headed with regard to targets being explored to vanquish the elusive neurodegenerative disease.

Original title: “AAIC 2026: Tau-targeted Alzheimer’s treatments heat up while amyloid therapies persist”

Neuropaediatric Rare Diseases (NRDs) impose a profound and multidimensional burden on patients, families, and healthcare systems. Persistently low clinical trial success rates reflect an unmet methodological need as much as a therapeutic one. A fundamental bottleneck is the absence of ... Neuropaediatric Rare Diseases (NRDs) impose a profound and multidimensional burden on patients, families, and healthcare systems. Persistently low clinical trial success rates reflect an unmet methodological need as much as a therapeutic one. A fundamental bottleneck is the absence of fit-for-purpose clinical outcome assessments. EAC is an employee of Engrail Therapeutics, Inc. and may hold stock options. EBK has received funding from numerous commercial entities (full list available in manuscript) to consult on trial design or conduct clinical or laboratory validation studies in genetic neurodevelopmental or neurodegenerative disorders; all funds are directed to RUMC in support of rare-disease programmes, with no personal remuneration and no relevant institutional financial interests. EAC is an employee of Engrail Therapeutics, Inc. and may hold stock options. EBK has received funding from numerous commercial entities (full list available in manuscript) to consult on trial design or conduct clinical or laboratory validation studies in genetic neurodevelopmental or neurodegenerative disorders; all funds are directed to RUMC in support of rare-disease programs, with no personal remuneration and no relevant institutional financial interests. Methodological challenges in outcomes research for early-trials for implementation of new therapies in neuropediatric rare diseases · Orphanet Journal of Rare Diseases, 20 (1) (2025), pp. 1-7, 10.1186/S13023-025-03814-0 ... J. Busner, G. Pandina, S. Day, A. Mahableshwarkar, L. Kempf, M. Sheean, J. Dunn · Patient Centricity: Design and Conduct of Clinical Trials in Orphan Diseases: Third of Three Sets of Expanded Proceedings from the 2020 ISCTM Autumn Conference on Pediatric Drug Development

Original title: “Outcome Strategies for Clinical Trials in Neuropaediatric Rare Diseases - ScienceDirect”

evaluation of anal cancer screening among 18 to 34 year old men who have sex with men living with hiv over a 10 year period

Original title: “Evaluation of Anal Cancer Screening Among 18-to-34-Year-Old Men Who Have Sex With Men Living With HIV Over a 10-Year Period.”

when candida ciferrii isn t candida sequencing reveals multidrug resistant blastobotrys adeninivorans pd peritonitis

Original title: “When "Candida ciferrii" Isn't Candida: Sequencing reveals multidrug-resistant Blastobotrys adeninivorans PD peritonitis.”

coronary artery bypass grafting for anomalous rca from lad with proximal lad stenosis

Original title: “Coronary Artery Bypass Grafting for Anomalous RCA From LAD With Proximal LAD Stenosis.”

primary high grade intraperitoneal sarcoma mimicking a germ cell tumor in an 8 year old girl in somaliland a diagnostic challenge

Original title: “Primary High-Grade Intraperitoneal Sarcoma Mimicking a Germ Cell Tumor in an 8-Year-Old Girl in Somaliland: A Diagnostic Challenge.”

Psychedelics are gaining momentum in depression, with one treating physician predicting that the drug class could “wipe out the SSRIs” if safety and durability hold up.

Original title: “Psychedelics surge again as Compass, Definium provide more clinical validation”