why promising atmps fail to reach patients a qualitative stakeholder informed analysis of cell therapy access in europe

Original title: “Why promising ATMPs fail to reach patients: a qualitative stakeholder-informed analysis of cell therapy access in Europe.”

dordaviprone for diffuse midline glioma a landmark approval or a premature step

Original title: “Dordaviprone for Diffuse Midline Glioma: A Landmark Approval or a Premature Step?”

machine learning derived speech index as a biomarker for huntington s disease severity

Original title: “A machine learning-derived speech index as a biomarker for Huntington's disease severity.”

addison s disease in italy mortality and survival by etiology

Original title: “Addison's disease in Italy: mortality and survival by etiology.”

msl2 disruption leads to autism like behaviors impairs neurogenesis and alters histone h4k16 acetylation

Original title: “MSL2 disruption leads to autism-like behaviors, impairs neurogenesis, and alters histone H4K16 acetylation.”

central nervous system tumors in xeroderma pigmentosum five cases and review of the literature

Original title: “Central Nervous System Tumors in Xeroderma Pigmentosum: Five Cases and Review of the Literature.”

primary isolated lumbar spine myeloid sarcoma a rare case report and review of the literature

Original title: “Primary isolated lumbar spine myeloid sarcoma: a rare case report and review of the literature.”

prospective multi center newborn screening for thalassemia by comprehensive analysis of thalassemia alleles catsa based on single molecule real time sequencing in guangxi china

Original title: “A Prospective Multi-Center Newborn Screening for Thalassemia by Comprehensive Analysis of Thalassemia Alleles (CATSA) Based on Single Molecule Real-Time Sequencing in Guangxi, China.”

saphenous vein graft kinking secondary to left atrial appendage clip causing type 5 myocardial infarction

Original title: “Saphenous Vein Graft Kinking Secondary to Left Atrial Appendage Clip Causing Type 5 Myocardial Infarction.”

factor structure and longitudinal measurement invariance of the psychopathology in autism checklist

Original title: “Factor Structure and Longitudinal Measurement Invariance of the Psychopathology in Autism Checklist.”

under enrollment in rare kidney disease trials lessons learned from the privent recurrent fsgs

Original title: “Under-enrollment in rare kidney disease trials: lessons learned from the PRIVENT recurrent FSGS.”

protocadherin 7 positive membranous nephropathy in china

Original title: “Protocadherin 7 Positive Membranous Nephropathy in China.”

synchronous isolated cystic pancreatic metastasis arising from primary colorectal cancer a rare case report

Original title: “Synchronous Isolated Cystic Pancreatic Metastasis Arising From Primary Colorectal Cancer: A Rare Case Report.”

clinical utility of telangiectasia quantification as markers of vascular disease progression in systemic sclerosis

Original title: “The clinical utility of telangiectasia quantification as markers of vascular disease progression in systemic sclerosis.”

cardiac tamponade secondary to massive pericardial effusion in severe primary hypothyroidism a case report

Original title: “Cardiac Tamponade Secondary to Massive Pericardial Effusion in Severe Primary Hypothyroidism: A Case Report.”

recent advances in imaging of demyelinating and dysmyelinating white matter lesions

Original title: “Recent Advances in Imaging of Demyelinating and Dysmyelinating White Matter Lesions.”

uric acid to hdl c ratio as a novel biomarker for sarcopenia a national study with machine learning insights

Original title: “Uric Acid to HDL-C Ratio as a Novel Biomarker for Sarcopenia: A National Study with Machine Learning Insights.”

shield a weakly supervised graph attention neural network for decoding disease relevant cell cell interactions

Original title: “SHIELD: A weakly supervised graph attention neural network for decoding disease-relevant cell-cell interactions.”