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11 p, 529.4 KB |
Nivolumab and sunitinib combination in advanced soft tissue sarcomas : A multicenter, single-arm, phase Ib/II trial
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Martin-Broto, J. (Universidad de Sevilla) ;
Hindi, Nadia (Universidad de Sevilla) ;
Grignani, G. (Candiolo Cancer Institute) ;
Martinez-Trufero, J. (Hospital Universitario Miguel Servet (Saragossa)) ;
Redondo, A. (Hospital Universitario La Paz (Madrid)) ;
Valverde, Claudia (Vall d'Hebron Institut d'Oncologia) ;
Stacchiotti, S. (Istituto Nazionale Tumori) ;
López Pousa, Antonio (Institut d'Investigació Biomèdica Sant Pau) ;
D'Ambrosio, L. (Candiolo Cancer Institute) ;
Gutierrez, A. (Hospital Universitari Son Espases (Palma de Mallorca, Balears)) ;
Perez-Vega, H. (Hospital Universitario Virgen del Rocío (Sevilla, Andalusia)) ;
Encinas-Tobajas, V. (Hospital Universitario Virgen del Rocío (Sevilla, Andalusia)) ;
De Álava, Enrique (Universidad de Sevilla) ;
Collini, Paola (Istituto Nazionale Tumori) ;
Peña-Chilet, M. (Hospital Virgen Del Rocio) ;
Dopazo, J. (Hospital Virgen Del Rocío) ;
Carrasco-Garcia, I. (Universidad de Sevilla) ;
Lopez-Alvarez, M. (Universidad de Sevilla) ;
Moura, D.S. (Universidad de Sevilla) ;
Lopez-Martin, Jose A (Instituto de Investigación Sanitaria Hospital 12 de Octubre (i+12)) ;
Universitat Autònoma de Barcelona
Sarcomas exhibit low expression of factors related to immune response, which could explain the modest activity of PD-1 inhibitors. A potential strategy to convert a cold into an inflamed microenvironment lies on a combination therapy. [...]
2020 -  10.1136/jitc-2020-001561
Journal for immunotherapy of cancer, Vol. 8 Núm. 2 (17 2020) , p. e001561
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12 p, 2.7 MB |
Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale
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Jacobs, M.B. (Pediatrics. Epidemiology. and Biostatistics. George Washington University) ;
James, Meredith K (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust. Central Parkway) ;
Lowes, Linda P (The Abigail Wexner Research Institute at Nationwide Children's Hospital) ;
Alfano, Lindsay N (The Abigail Wexner Research Institute at Nationwide Children's Hospital) ;
Eagle, M. (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust. Central Parkway) ;
Muni Lofra, R. (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust. Central Parkway) ;
Moore, U. (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust. Central Parkway) ;
Feng, J. (Center for Translational Science. Division of Biostatistics and Study Methodology. Children's National Health System) ;
Rufibach, L.E. (The Jain Foundation) ;
Rose, K. (The Children's Hospital at Westmead. The University of Sydney) ;
Duong, T. (Lucile Salter Packard Children's Hospital at Stanford) ;
Bello, Luca (Department of Neuroscience. University of Padova) ;
Pedrosa-Hernández, Irene (Institut d'Investigació Biomèdica Sant Pau) ;
Holsten, Scott (Neuroscience Institute. Carolinas Neuromuscular/ALS-MDA Center. Carolinas HealthCare System) ;
Sakamoto, C. (Department of Physical Rehabilitation. National Center Hospital. National Center of Neurology and Psychiatry) ;
Canal, Aurélie (Institut de Myologie. AP-HP. GH Pitié-Salpêtrière) ;
Sanchez-Aguilera Práxedes, N. (Hospital Universitario Virgen del Rocío (Sevilla, Andalusia)) ;
Thiele, S. (Friedrich-Baur-Institute. Department of Neurology. Ludwig-Maximilians University of Munich) ;
Siener, C. (Department of Neurology Washington University School of Medicine) ;
Vandevelde, B. (Service des Maladies Neuromusculaire et de la SLA. Hôpital de La Timone) ;
DeWolf, Brittney (Cooperative International Neuromuscular Research Group (CINRG). Children's National Health System) ;
Maron, E. (ELAN-PHYSIO. Praxis für Physiotherapie Maron) ;
Guglieri, M. (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust. Central Parkway) ;
Hogrel, J.Y. (Institut de Myologie (París, França)) ;
Blamire, Andrew (Magnetic Resonance Centre. Institute for Cellular Medicine. Newcastle University) ;
Carlier, Pierre G (AIM & CEA NMR Laboratory. Institute of Myology. Pitié-Salpêtrière University Hospital) ;
Spuler, S. (Charite Muscle Research Unit. Experimental and Clinical Research Center. a joint cooperation of the Charité Medical Faculty and the Max Delbrück Center for Molecular Medicine) ;
Day, John W (Department of Neurology and Neurological Sciences. Stanford University School of Medicine) ;
Jones, K.J. (The Children's Hospital at Westmead. The University of Sydney) ;
Bharucha-Goebel, Diana (National Institutes of Health (Bethesda, Estats Units d'Amèrica)) ;
Salort-Campana, E. (Service des Maladies Neuromusculaire et de la SLA. Hôpital de La Timone) ;
Pestronk, Alan (Washington University School of Medicine) ;
Walter, Maggie C (Friedrich-Baur-Institute. Department of Neurology. Ludwig-Maximilians University of Munich) ;
Paradas, C. (Instituto de Biomedicina de Sevilla) ;
Stojkovic, T. (Institut de Myologie. AP-HP. GH Pitié-Salpêtrière) ;
Mori-Yoshimura, Madoka (National Center of Neurology and Psychiatry Tokyo) ;
Bravver, Elena (Neuroscience Institute. Carolinas Neuromuscular/ALS-MDA Center. Carolinas HealthCare System) ;
Diaz-Manera, Jordi (Institut d'Investigació Biomèdica Sant Pau) ;
Pegoraro, Elena (University of Padova) ;
Mendell, J. R. (The Abigail Wexner Research Institute at Nationwide Children's Hospital) ;
Mayhew, A.G. (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust. Central Parkway) ;
Straub, Volker (The John Walton Muscular Dystrophy Research Centre. Translational and Clinical Research Institute. Newcastle University and Newcastle Hospitals NHS Foundation Trust. Central Parkway)
Objective: Dysferlinopathy is a muscular dystrophy with a highly variable clinical presentation and currently unpredictable progression. This variability and unpredictability presents difficulties for prognostication and clinical trial design. [...]
2021 - 10.1002/ana.26044
Annals of neurology, Vol. 89 Núm. 5 (may 2021) , p. 967-978
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15 p, 807.5 KB |
Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe : methodological uncertainties
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Pontes García, Caridad (Parc Taulí Hospital Universitari. Institut d'Investigació i Innovació Parc Taulí (I3PT)) ;
Fontanet Sacristán, Juan Manuel (Institut d'Investigació Biomèdica Sant Pau) ;
Vives, Roser (Parc Taulí Hospital Universitari. Institut d'Investigació i Innovació Parc Taulí (I3PT)) ;
Sancho, Aranzazu (Instituto de Investigación Sanitaria Puerta de Hierro - Segovia de Arana) ;
Gómez-Valent, Mònica (Parc Taulí Hospital Universitari. Institut d'Investigació i Innovació Parc Taulí (I3PT)) ;
Ríos, José (Institut d'Investigacions Biomèdiques August Pi i Sunyer) ;
Morros, Rosa (Institut Universitari d'Investigació en Atenció Primària Jordi Gol) ;
Martinalbo, Jorge (Universitat Autònoma de Barcelona. Departament de Farmacologia, de Terapèutica i de Toxicologia) ;
Posch, Martin (Medical University of Vienna) ;
Koch, Armin (Hannover Medical School) ;
Roes, Kit (University of Utrecht, Utrecht, The Netherlands) ;
Rengerink, Katrien Oude (University of Utrecht, Heidelberglaan, CX Utrecht, The Netherlands) ;
Torrent-Farnell, Josep (Institut d'Investigació Biomèdica Sant Pau) ;
Torres, Ferran (Institut d'Investigacions Biomèdiques August Pi i Sunyer)
To assess uncertainty in regulatory decision-making for orphan medicinal products (OMP), a summary of the current basis for approval is required; a systematic grouping of medical conditions may be useful in summarizing information and issuing recommendations for practice. [...]
2018 - 10.1186/s13023-018-0926-z
Orphanet Journal of Rare Diseases, Vol. 13 (november 2018)
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14 p, 1.3 MB |
Infections in Dupilumab Clinical Trials in Atopic Dermatitis : A Comprehensive Pooled Analysis
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Eichenfield, Lawrence (Division of Pediatric and Adolescent Dermatology. Rady Children's Hospital) ;
Bieber, T. (Department of Dermatology and Allergy. Christine Kühne-Center for Allergy Research and Education. University of Bonn) ;
Beck, Lisa (University of Rochester Medical Center. Department of Dermatology) ;
Simpson, E. L. (Oregon Health and Science University. Department of Dermatology) ;
Thaçi, Diamant (Universitätsklinikum Schleswig-Holstein (Alemanya)) ;
de Bruin-Weller, Marjolein (University Medical Center Utrecht) ;
Deleuran, Mette (Aarhus University Hospital (Aarhus, Dinamarca)) ;
Silverberg, J. I. (Northwestern University Feinberg School of Medicine) ;
Ferrándiz, Carlos (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol) ;
Fölster-Holst, R. (University Hospital Schleswig-Holstein. Dermatology) ;
Chen, Z. (Regeneron Pharmaceuticals. Inc.) ;
Graham, N. M. H. (Regeneron Pharmaceuticals. Inc.) ;
Pirozzi, G. (Sanofi) ;
Akinlade, Bolanle (Regeneron Pharmaceuticals. Inc.) ;
Yancopoulos, G. D. (Regeneron Pharmaceuticals. Inc.) ;
Ardeleanu, Marius (Regeneron Pharmaceuticals. Inc.) ;
Universitat Autònoma de Barcelona
Background: Patients with moderate-to-severe atopic dermatitis (AD) have increased infection risk, including skin infections and systemic infections. Immunomodulators (e. g. , anti-tumor necrosis factors, anti-interleukin [anti-IL]-23, anti-IL-17, Janus kinase inhibitors) increase risk of infections. [...]
2019 - 10.1007/s40257-019-00445-7
American Journal of Clinical Dermatology, Vol. 20 Núm. 3 (january 2019) , p. 443-456
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14 p, 1.0 MB |
A new cognitive evaluation battery for Down syndrome and its relevance for clinical trials
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Sola, Susana de (Institut Hospital del Mar d'Investigacions Mèdiques) ;
Torre Fornell, Rafael de la (Centro de Investigación Biomédica en Red de Fisiopatología de la Obesidad y Nutrición) ;
Sánchez-Benavides, Gonzalo (Institut Hospital del Mar d'Investigacions Mèdiques) ;
Benejam, Bessy (Fundació Catalana Síndrome de Down) ;
Cuenca-Royo, Aida (Institut Hospital del Mar d'Investigacions Mèdiques) ;
Hoyo Soriano, Laura del (Institut Hospital del Mar d'Investigacions Mèdiques) ;
Rodríguez, Joan (Institut Hospital del Mar d'Investigacions Mèdiques) ;
Catuara-Solarz, Silvina (Centre de Regulació Genòmica) ;
Sánchez-Gutiérrez, Judit (Fundació Espai Salut (Barcelona)) ;
Dueñas-Espin, Ivan (Institut Hospital del Mar d'Investigacions Mèdiques) ;
Hernandez, Gimena (Institut Hospital del Mar d'Investigacions Mèdiques) ;
Peña-Casanova, J. (Jordi) (Institut Hospital del Mar d'Investigacions Mèdiques) ;
Langohr, Klaus (Institut Hospital del Mar d'Investigacions Mèdiques) ;
Videla, Sebastián (Fundació Catalana Síndrome de Down) ;
Blehaut, Henry (Fondation Jérôme Lejeune (Paris, França)) ;
Farre, Magi (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol) ;
Dierssen, Mara (Centro de Investigación Biomédica en Red de Enfermedades Raras) ;
TESDAD Study Group ;
Universitat Autònoma de Barcelona
Abstract: The recent prospect of pharmaceutical interventions for cognitive impairment of Down syndrome (DS) has boosted a number of clinical trials in this population. However, running the trials has raised some methodological challenges and questioned the prevailing methodology used to evaluate cognitive functioning of DS individuals. [...]
2015 - 10.3389/fpsyg.2015.00708
Frontiers in psychology, Vol. 6 Núm. 708 (June 2015)
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