320 results match your criteria: "and University of Paris[Affiliation]"

Computational modeling of superparamagnetic nanoparticle-based (affinity) diagnostics.

Front Bioeng Biotechnol

December 2024

Vascularized Composite Allotransplantation Laboratory, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States.

Introduction: Magnetic nanoparticles (MNPs), particularly iron oxide nanoparticles (IONPs), are renowned for their superparamagnetic behavior, allowing precise control under external magnetic fields. This characteristic makes them ideal for biomedical applications, including diagnostics and drug delivery. Superparamagnetic IONPs, which exhibit magnetization only in the presence of an external field, can be functionalized with ligands for targeted affinity diagnostics.

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Neuropsychological profile of French adults with early-treated phenylketonuria: a multicenter study.

J Neurol

December 2024

Service de Médecine Interne, Centre de Référence Des Maladies Héréditaires du Métabolisme, UMR INSERM 1253 « iBraiN », Université de Tours, CHU de Tours, Tours, France.

Background And Objective: Adult patients with early-treated phenylketonuria (AwET-PKU) may present some subtle neurocognitive deficits. The aim of the study was to investigate 1) neurocognitive functions in a large group of AwET-PKU 2) the influence of plasma phenylalanine (Phe).

Methods: Participants: 187 AwET-PKU (classic PKU [cPKU] 81%, mild PKU [mPKU] 14%, and mild persistent hyperphenylalaninemia [MPH] 5%).

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Reply to: comparing the efficacy of intravenous lidocaine versus placebo for postoperative analgesia in paediatric patients: methodology is important.

Eur J Anaesthesiol

January 2025

From the Department of Anaesthesia and Intensive Care, Robert Debré University Hospital, Assistance Publique des Hôpitaux de Paris (PP, SD) and University of Paris-Cité (PP, SD), FHU I2D2. Robert Debré University Hospital, Paris, France (PP, SD).

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Development of a pediatric oral solution of ONC201 using nicotinamide to enhance solubility and stability.

Int J Pharm

December 2024

Department of clinical pharmacy, Gustave Roussy Cancer Campus, Villejuif 94800, France; Université Paris-Saclay, CNRS, Institut des Sciences Moléculaires d'Orsay, Orsay 91405, France.

Diffuse intrinsic pontine glioma (DIPG) poses a significant treatment challenge in pediatric patients due to its aggressive nature and difficulty in crossing the blood-brain barrier with effective therapies. ONC201 (dordaviprone) shows promises in inducing apoptosis in cancer cells but suffers from poor water solubility and stability issues. Moreover, conventional solubilizing agents acceptable in formulations intended for adult patients are not suitable for pediatric use.

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Article Synopsis
  • The study aimed to investigate how common mental health issues and multiple disorders (multimorbidity) are among a large sample of French adults, focusing on women and men.
  • Data analysis of over 33,000 participants revealed that 40.6% reported at least one mental disorder, with a higher prevalence of disorders among women compared to men.
  • The research highlights specific patterns, such as overweight and smoking habits among men with multiple disorders, suggesting implications for public health strategies and further studies on mental health.
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Diagnostic and Therapeutic Insights Into Pediatric Neurosarcoidosis: Observations From French Pediatric Rheumatology Centers.

Pediatr Neurol

January 2025

Department of General Pediatrics, Pediatric Internal Medicine, Rheumatology and Infectious Diseases, National Reference Centre for Rare Pediatric Inflammatory Rheumatisms and Systemic Autoimmune Diseases (RAISE), Robert-Debré University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Université Paris Cité, INSERM, Centre de Recherche sur l'inflammation UMR 1149, Paris, France. Electronic address:

Article Synopsis
  • - The study reviewed 11 pediatric patients diagnosed with neurosarcoidosis (NS), predominantly affecting girls, with an average diagnosis age of around 10 to 11.5 years, highlighting typical neurological symptoms such as headaches and eye involvement.
  • - Most patients experienced meningitis and were treated primarily with corticosteroids and TNF-alpha inhibitors, with eight out of 11 requiring biologic therapies to achieve remission.
  • - The findings emphasize the importance of recognizing the clinical features of pediatric NS and suggest that early intervention with TNF-alpha biologics can lead to better management outcomes for these children.
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CPX-351 has been approved for patients with therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (MRC-AML). No extensive data are available on MRD and long-term clinical outcome using CPX-351 in AML in real-life. We retrospectively collected data from 168 patients in 36 centers in France and Italy who had received one or two cycles of induction with CPX-351.

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Article Synopsis
  • - The standard method for assessing metabolic dysfunction-associated steatosis in clinical trials is by evaluating liver biopsies on glass slides; however, shipping these slides poses logistical challenges and risks of damage.
  • - This study found that using digital images on the AISight whole slide image management system for assessing steatohepatitis offers comparable accuracy to traditional glass slide evaluations, with both methods verified against a consensus score from expert pathologists.
  • - The results showed that the agreement between digital scoring and the established "ground truth" was not inferior to that of glass scoring, indicating that digital assessments can reliably replace traditional methods in clinical trial settings for liver disease.
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A Phase 2 Randomized Trial of Survodutide in MASH and Fibrosis.

N Engl J Med

July 2024

From the Stravitz-Sanyal Institute for Liver Disease and Metabolic Health, Virginia Commonwealth University School of Medicine, Richmond (A.J.S.); Liverpat and University of Paris (P.B.), and Sorbonne Université, Institute for Cardiometabolism and Nutrition, Assistance Publique-Hôpitaux de Paris, INSERM Unité Mixte de Recherche Scientifique 1138, Centre de Recherche des Cordeliers (V.R.) - all in Paris; Boehringer Ingelheim, Ingelheim am Rhein (M.F., S.A.H and R.Y.), Saarland University Medical Center, Homburg (J.M.S.), and University of the Saarland, Saarbrücken (J.M.S.) - all in Germany; Covenant Metabolic Specialists, Sarasota, FL (G.W.N.); Houston Methodist Hospital and Houston Research Institute, Houston (M.N.), and the Texas Liver Institute, University of Texas Health San Antonio, San Antonio (E.L. and N.A.) - all in Texas; the University of Turin, Turin, Italy (E.B.); the Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle University, and Newcastle National Institute for Health and Care Research Biomedical Research Centre, Newcastle upon Tyne Hospitals National Health Service (NHS) Foundation Trust, Newcastle upon Tyne (Q.M.A.), the University Hospitals Birmingham NHS Foundation Trust and the University of Birmingham, Birmingham (P.N.N.), and the Institute of Hepatology, Faculty of Life Sciences and Medicine, King's College London and King's College Hospital, London (P.N.N.) - all in the United Kingdom; Boehringer Ingelheim Pharmaceuticals, Ridgefield, CT (A.H.-T.); and Arizona Liver Health, Chandler (N.A.).

Background: Dual agonism of glucagon receptor and glucagon-like peptide-1 (GLP-1) receptor may be more effective than GLP-1 receptor agonism alone for treating metabolic dysfunction-associated steatohepatitis (MASH). The efficacy and safety of survodutide (a dual agonist of glucagon receptor and GLP-1 receptor) in persons with MASH and liver fibrosis are unclear.

Methods: In this 48-week, phase 2 trial, we randomly assigned adults with biopsy-confirmed MASH and fibrosis stage F1 through F3 in a 1:1:1:1 ratio to receive once-weekly subcutaneous injections of survodutide at a dose of 2.

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Article Synopsis
  • This study aimed to evaluate the effectiveness and safety of two procedures—peroral endoscopic myotomy with septotomy (POEM+S) and without septotomy (POEM-S)—in treating symptomatic epiphrenic diverticula (ED) alongside motility disorders.
  • Conducted across 21 international centers from 2014 to 2023, the study included 85 patients, measuring clinical success primarily through the Eckardt score.
  • Results indicated that both procedures had similar clinical success rates (83% for POEM+S vs. 86.8% for POEM-S), technical success, and adverse event rates, but POEM-S patients had a longer hospital stay.
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Methionine synthase reductase deficiency (cblE) is a rare autosomal recessive inborn error of cobalamin metabolism caused by pathogenic variants in the methionine synthase reductase gene (). Patients usually exhibit early-onset bone marrow failure with pancytopenia including megaloblastic anemia. The latter can remain isolated or patients may present developmental delay and rarely macular dysfunction.

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Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura.

N Engl J Med

May 2024

From the Department of Haematology, University College London Hospitals, London (M.S.); the Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta (A.A.); the Department of Internal Medicine, Ohio State University, Columbus (S.R.C.); the Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris (AP-HP) and Sorbonne Université (P.C.), the Department of Pediatric Nephrology, Robert Debré Hospital, AP-HP and University of Paris (C.D.), and the Department of Pediatric Nephrology, Hôpital Universitaire Necker-Enfants Malades, AP-HP (N.B.) - all in Paris; the Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg (W.-A.H.), and the Section of Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Universitätsklinikum Jena, Jena (K.K.) - both in Germany; the Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna (P.K.); the Department of Hematology and Central Hematologic Laboratory, Bern University Hospital, University of Bern, Bern, Switzerland (J.A.K.H.); the Hematology and Hemotherapy Service, Mother and Child Hospital, Biomedical Research Institute of A Coruña, University Hospital Complex of A Coruña, A Coruña, Spain (M.F.L.-F.); the Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan (M.M.); the Division of Hematology, Department of Medicine, and Department of Pathology, Duke University, Durham, NC (T.L.O.); the Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); and Takeda Development Center Americas, Cambridge, MA (I.B., M.C., H.L., B.M., M.P., P.P, S.X., P.Z., L.T.W.).

Article Synopsis
  • Congenital thrombotic thrombocytopenic purpura (TTP) is caused by a severe deficiency of the ADAMTS13 enzyme, and the study compared the effectiveness of recombinant ADAMTS13 to standard therapies in preventing TTP events in patients.
  • The phase 3 trial involved patients receiving either recombinant ADAMTS13 or standard therapy in alternating 6-month periods, measuring outcomes like acute TTP events, safety, and pharmacokinetics.
  • Results showed no acute TTP events during recombinant ADAMTS13 prophylaxis, whereas standard therapy had one event; adverse events were lower with recombinant ADAMTS13, and no patients discontinued due to side effects from it, indicating it may be
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Lessons from prospective longitudinal follow-up of a French APECED cohort.

J Clin Endocrinol Metab

April 2024

Department of Endocrinology, Diabetology and Metabolism, Huriez Hospital, Lille University Hospital, F-59000 Lille, France.

Article Synopsis
  • APECED syndrome is a rare genetic disorder caused by mutations in the AIRE gene, typically characterized by a triad of symptoms including hypoparathyroidism, adrenal failure, and chronic mucocutaneous candidiasis (CMC), along with other non-endocrine issues.
  • In a national study involving 25 patients from 23 families, researchers identified 11 different variants of the AIRE gene, including two previously unreported variants, and found that a majority of patients displayed multiple clinical manifestations.
  • The study revealed significant immunological disturbances, such as NK cell lymphopenia and altered B lymphocyte homeostasis, and highlighted a variety of non-endocrine symptoms that could potentially be life-threatening, emphasizing the need for
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Impact of direct use of artificial intelligence algorithms on patient autonomy in dermatology.

Ann Dermatol Venereol

March 2024

Dermatology Department and University of Paris, Saint-Louis Hospital, Paris, France; Membre du Groupe d'Ethique en Dermatologie. Electronic address:

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Background: Although rotavirus vaccination has reduced the global burden of the virus, morbidity and mortality from rotavirus infection remain high in Sub-Saharan Africa. This study aimed to determine the prevalence of rotavirus and adenovirus infections in children under five years with acute gastroenteritis and to identify factors associated with rotavirus infection after the introduction of the Rotasiil® vaccine in 2019 in Kisangani, Democratic Republic of the Congo (DRC).

Methods: This study consisted of a cross-sectional hospital-based survey conducted from May 2022 to April 2023 in four health facilities in Kisangani, using a fecal-based test (rapid antigenic immuno-chromatographic diagnostic test, BYOSYNEX adenovirus/rotavirus BSS, Biosynex SA, Illkirch-Graffenstaden, France) of rotavirus and adenovirus infections among children under five years of age with acute gastroenteritis.

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A Phase 3, Randomized, Controlled Trial of Resmetirom in NASH with Liver Fibrosis.

N Engl J Med

February 2024

From the University of Oxford, Oxford (S.A.H.), the Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle University, Newcastle upon Tyne (Q.M.A.), and the National Institute for Health Research, Biomedical Research Centre at University Hospitals Birmingham NHS Foundation Trust and the University of Birmingham, Birmingham (P.N.N.) - all in the United Kingdom; Pinnacle Clinical Research, San Antonio (S.A.H., M.R.), South Texas Research Institute, Edinburg (R.P.), and Houston Methodist Hospital, Houston Research Institute, Houston (M.N.) - all in Texas; Liverpat and University of Paris (P.B.), INSERM, Unité Mixte de Recherche Scientifique (UMRS) 1139, Centre de Recherche sur l'Inflammation (L.C.), and Sorbonne Université, ICAN Institute for Cardiometabolism and Nutrition, Assistance Publique-Hôpitaux de Paris (APHP), INSERM, UMRS 1138, Centre de Recherche des Cordeliers (V.R.), Paris, and Université Paris-Cité, Department of Hepatology, Beaujon Hospital, APHP, Clichy (L.C.) - all in France; Duke University Health System, Durham, NC (C.D.G.); the Metabolic Liver Research Program, I. Department of Medicine, University Medical Center of Johannes Gutenburg University Mainz, Mainz (J.M.S.), the Department of Internal Medicine II, Saarland University Medical Center, Homburg (J.M.S.), and Klinikum St. Georg Leipzig, Leipzig (I.S.) - all in Germany; MASLD Research Center, the Division of Gastroenterology and Hepatology, University of California, San Diego, La Jolla (R.L.); Madrigal Pharmaceuticals, West Conshohocken, PA (R.T., D.L.); University of Arizona for Medical Sciences (S.E.M.) and Arizona Liver Health (N.A.) - both in Tucson; Covenant Metabolic Specialists, Sarasota (G.W.N.), and Flourish Research, Boca Raton (S.J.B.) - both in Florida; the Transplant Institute, Department of Medicine, University of Chicago Pritzker School of Medicine, and the Transplant Institute, Center for Liver Diseases, University of Chicago Biological Sciences - both in Chicago (M.E.R., M.R.C.); the Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, MN (M.F.A.); the Department of Medicine, Inova Fairfax Medical Campus, Falls Church (Z.Y.), and Virginia Commonwealth University, Richmond (A.J.S.) - both in Virginia; the Department of Gastroenterology and Hepatology, Antwerp University Hospital, Edegem (S.F.), and Cliniques Universitaires Saint-Luc, Service d'Hépato-gastroentérologie, UCLouvain, Brussels (N.L.) - both in Belgium; the Liver Unit at the IRCCS "Casa Sollievo della Sofferenza" Hospital, San Giovanni Rotondo, Italy (A.M.); the Liver Unit, Vall d'Hebron University Hospital, Vall d'Hebron Institut de Recerca, Universitat Autònoma de Barcelona, Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas, Barcelona (J.M.P.); and the Division of Liver Diseases, Icahn School of Medicine at Mt. Sinai, New York (M.B.B.).

Article Synopsis
  • * A phase 3 trial is underway, where adult participants with confirmed NASH are randomly assigned to receive either resmetirom (80 mg or 100 mg) or a placebo, with primary goals of NASH resolution and improvement in fibrosis after 52 weeks.
  • * In the trial's results, a significantly higher percentage of patients taking resmetirom experienced NASH resolution and fibrosis improvement compared to those on placebo, along with notable reductions in cholesterol levels, although some participants did report diarrhea.
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Article Synopsis
  • Researchers developed a mouse model to better understand NS progression by analyzing molecular profiles that match both mice and human patients.
  • The study highlights the role of specific cytokines and proteases in disease severity and proposes a new signaling pathway involving tissue kallikrein-related proteases and IL-36, offering potential therapeutic targets.
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Tissue engineering strategies for breast reconstruction: a literature review of current advances and future directions.

Ann Transl Med

February 2024

Vascularized Composite Allotransplantation Laboratory, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.

Background And Objective: Mastectomy is a primary treatment for breast cancer patients, and both autologous and implant-based reconstructive techniques have shown excellent results. In recent years, advancements in bioengineering have led to a proliferation of innovative approaches to breast reconstruction. This article comprehensively explores the promising perspectives offered by bioengineering and tissue engineering in the field of breast reconstruction.

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Article Synopsis
  • The Ritux 3 trial showed that rituximab is effective and safe as a first-line treatment for pemphigus, although long-term follow-up data was previously lacking.!* -
  • This 7-year follow-up study evaluated the long-term outcomes for patients treated with rituximab alongside prednisone versus those treated with prednisone alone, focusing on disease-free survival and relapse rates.!* -
  • Results indicated that 93% of patients in the rituximab group achieved complete remission without corticosteroids, showing significantly higher disease-free survival and lower relapse rates compared to the prednisone-only group.!*
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Background: In two phase 3 trials for first-line therapy in adults with HIV-1, doravirine showed non-inferior efficacy, a favourable safety profile, and a superior lipid profile to darunavir and efavirenz through to 48 and 96 weeks. Here we report 192-week results from both studies.

Methods: DRIVE-FORWARD and DRIVE-AHEAD are multicentre, double-blind, randomised, active comparator-controlled, phase 3 trials of first-line antiretroviral treatment in adults with HIV-1.

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Environmental pressures and pesticide exposure associated with an increase in the share of plant-based foods in the diet.

Sci Rep

November 2023

Nutritional Epidemiology Research Team (EREN), Epidemiology and Statistics Research Center (CRESS), Sorbonne Paris Nord University and University of Paris, Inserm, INRAE, Cnam, 74 Rue Marcel Cachin, 93017, Bobigny, France.

Diets rich in plant-based foods are encouraged for human health and to preserve resources and the environment but the nutritional quality and safety of such diets is debated. This study aimed to model nutritionally adequate diets with increasing plant food content and to characterise the derived diets using a multicriteria approach including, nutrients intake, environmental pressures and exposure to pesticides. Using data of the NutriNet-Santé cohort (N = 29,413), we implemented stepwise optimization models to identified maximum plant-food content under nutritional constraints.

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What distinguishes endoscopic submucosal dissection (ESD) from endoscopic mucosal resection is the need for three foot pedals to activate the electrosurgical unit, flushing and knife injection. The lack of connection between the various pedals of different shapes and brands leads to numerous pedals displacements and potential mistakes. The aim of this study was to evaluate an Innovative PEdal FIXator (IPEFIX) to reduce pedal mistakes during ESD.

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