SUBREGISTRO PROSPECTIVO, MULTICÉNTRICO Y OBSERVACIONAL DE SEGURIDAD POSTAUTORIZACIÓN PARA CARACTERIZAR EL PERFIL DE SEGURIDAD A LARGO PLAZO DEL USO COMERCIAL DE ELIGLUSTAT (CERDELGA®) EN PACIENTES ADULTOS CON ENFERMEDAD DE GAUCHER.

Dades bàsiques

Protocol:
OBS14099
EURDRACT:
NCT:
Centre:
HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
Any inici:
2018
Any de finalització:
ESTUDIO OBSERVACIONAL INTERNACIONAL

Documents

  • No hi ha documents

Participants

Grups d'Investigació

Finançadors - Promotors

SANOFI GENZYME

Resultats de l'Assaig Clínic


Angiotensin-Converting Enzyme Inhibitors and Angiotensin Receptor Blockers Withdrawal Is Associated with Higher Mortality in Hospitalized Patients with COVID-19

Roy-Vallejo, Emilia; (...); Millan Nunez-Cortes, Jesus

Article. 10.3390/jcm10122642. 2021

  • Open Access.

ASSOCIATION OF HYPERTENSION WITH ALL-CAUSE MORTALITY AMONG HOSPITALIZED PATIENTS WITH COVID-19

Rodilla, E; (...); Gomez-Huelgas, R

Meeting Abstract. 2021

  • Open Access.

Association of Hypertension with All-Cause Mortality among Hospitalized Patients with COVID-19.

Rodilla E; (...); Gómez-Huelgas R

Article. 10.3390/jcm9103136. 2020

  • Open Access.

Autoimmune Diseases and COVID-19 as Risk Factors for Poor Outcomes: Data on 13,940 Hospitalized Patients from the Spanish Nationwide SEMI-COVID-19 Registry

Gutierrez, MDA; (...); Huelgas, RG

Article. 10.3390/jcm10091844. 2021

  • Open Access.

Biomarkers response to anti-TNF treatment in Crohn's disease through the intestinal microbiota

Sanchis, L; (...); Cortes, X

Meeting Abstract. 2020


Biomonitoring of polychlorinated dibenzo-p-dioxins (PCDDs), polychlorinated dibenzofurans (PCDFs) and dioxin-like polychlorinated biphenyls (dl-PCBs) in human milk: Exposure and risk assessment for lactating mothers and breastfed children from Spain

Hernandez, CS; (...); BETTERMILK

Article. 10.1016/j.scitotenv.2020.140710. 2020


Breastfeeding and Inborn Errors of Amino Acid and Protein Metabolism: A Spreadsheet to Calculate Optimal Intake of Human Milk and Disease-Specific Formulas.

Vitoria-Miñana I; (...); Correcher-Medina P

Article. 10.3390/nu15163566. 2023

  • Open Access.

Carbohydrate status in patients with phenylketonuria

Couce, ML; (...); Leis, R

Article. 10.1186/s13023-018-0847-x. 2018

  • Open Access.

Clinical features and health-related quality of life in adult patients with mucopolysaccharidosis IVA: the Spanish experience.

Quijada-Fraile, P; (...); Morales Conejo M

Article. 10.1186/s13023-021-02074-y. 2021

  • Open Access.

Clinical features and risk factors for mortality among long-term care facility residents hospitalized due to COVID-19 in Spain.

Ramos-Rincón JM; (...); SEMI-COVID-19 Network

Article. 10.1093/gerona/glab305. 2021

  • Open Access.

Clinical Outcomes of Patients with Chronic Neuropathic Form of Gaucher Disease in the Spanish Real-World Setting: A Retrospective Study.

Stanescu, Sinziana; (...); O'Callaghan Gordo, Mar

Article. 10.3390/biomedicines11102861. 2023

  • Open Access.

Correction: Vitamin C and folate status in hereditary fructose intolerance.

Cano A; (...); de Las Heras J

Correction. 10.1038/s41430-023-01334-3. 2023

  • Open Access.

Current HHT genetic overview in Spain and its phenotypic correlation: data from RiHHTa registry

Sanchez-Martinez, R; (...); Riera-Mestre, A

Article. 10.1186/s13023-020-01422-8. 2020

  • Open Access.

Descriptive study of patients with Gaucher disease type 3 in Spain and clinical response to different therapies

Giraldo, Pilar; (...); Visa-Rene, Nuria

Meeting Abstract. 10.1016/j.ymgme.2023.107843. 2024


DEVELOPMENT AND VALIDATION OF A MCDA FRAMEWORK FOR EVALUATION AND DECISION-MAKING OF ORPHAN DRUGS IN SPAIN

Poveda, J. L.; (...); Vitoria, I

Meeting Abstract. 2019


Development and validation of an MCDA framework for evaluation and decision-making of orphan drugs in Spain

Badia, X; (...); Poved, JL

Article. 10.1080/21678707.2019.1652163. 2019


Dietary practices in methylmalonic acidaemia: a European survey.

Pinto A; (...); MacDonald A

Article. 10.1515/jpem-2019-0277. 2020

  • Open Access.

Dietary practices in propionic acidemia: A European survey.

Daly, A; (...); MacDonald, A

Article. 10.1016/j.ymgmr.2017.09.002. 2017

  • Open Access.

Does admission acetylsalicylic acid uptake in hospitalized COVID-19 patients have a protective role? Data from the Spanish SEMI-COVID-19 Registry.

Formiga F; (...); SEMI-COVID-19 SEMI-COVID-19 Network

Article. 10.1007/s11739-021-02870-1. 2021

  • Open Access.

Early feeding practices in infants with phenylketonuria across Europe

Pinto, A; (...); MacDonald, A

Article. 10.1016/j.ymgmr.2018.07.008. 2018

  • Open Access.

Healthcare workers hospitalized due to COVID-19 have no higher risk of death than general population. Data from the Spanish SEMI-COVID-19 Registry.

Díez-Manglano J; (...); SEMI-COVID-19 Network

Article. 10.1371/journal.pone.0247422. 2021

  • Open Access.

Identification of Clinical Variants beyond the Exome in Inborn Errors of Metabolism.

Soriano-Sexto, Alejandro; (...); Perez, Belen

Article. 10.3390/ijms232112850. 2022

  • Open Access.

Implementation and results of a risk-sharing scheme for enzyme replacement therapy in lysosomal storage diseases

Edo-Solsona, MD, Vitoria-Minana, I, Poveda-Andres, JL

Article. 10.7399/fh.11262. 2020


Integration of multi-omics layers empowers precision diagnosis through unveiling pathogenic mechanisms on maple syrup urine disease.

Tejedor, Juan Ramon; (...); Perez, Belen

Article. 10.1002/jimd.12829. 2024

  • Open Access.

Lactose and Galactose Content in Spanish Cheeses: Usefulness in the Dietary Treatment of Patients with Galactosaemia.

Vitoria, Isidro; (...); Garcia-Peris, Monica

Article. 10.3390/nu15030594. 2023

  • Open Access.

Lymphocyte Medium-Chain Acyl-CoA Dehydrogenase Activity and Its Potential as a Diagnostic Confirmation Tool in Newborn Screening Cases.

Alcaide, Patricia; (...); Perez, Belen

Article. 10.3390/jcm11102933. 2022

  • Open Access.

New insights in the TRAZELGA project for the adult type 1 Gaucher disease patients treated with eliglustat follow-up

Gonzalo, IS; (...); Castellano, PG

Meeting Abstract. 10.1016/j.ymgme.2020.12.239. 2021

  • Open Access.

New variants expand the neurological phenotype of COQ7 deficiency.

Fabra, Maria Alcazar; (...); Santos-Ocana, Carlos

Article. 10.1002/jimd.12776. 2024

  • Open Access.

Newly validated biomarkers of brain damage may shed light into the role of oxidative stress in the pathophysiology of neurocognitive impairment in dietary restricted phenylketonuria patients

Rausell, Dolores; (...); Chafer-Pericas, Consuelo

Review. 10.1038/s41390-018-0202-x. 2019

  • Open Access.

Nutritional Management of Patients with Fatty Acid Oxidation Disorders.

Pena-Quintana, Luis, Correcher-Medina, Patricia

Article. 10.3390/nu16162707. 2024

  • Open Access.

Nutritional status in patients with protein metabolism disorders. Case-control study.

Robredo Garcia, Isidro; (...); Martinez Costa, Cecilia

Article. 10.1016/j.anpede.2024.10.012. 2024

  • Open Access.

Prior Treatment with Statins is Associated with Improved Outcomes of Patients with COVID-19: Data from the SEMI-COVID-19 Registry

Torres-Pena, JD; (...); Gomez-Huelgas, R

Article. 10.1007/s40265-021-01498-x. 2021

  • Open Access.

Prognostic Value of D-dimer to Lymphocyte Ratio (DLR) in Hospitalized Coronavirus Disease 2019 (COVID-19) Patients: A Validation Study in a National Cohort

Oblitas, Crhistian-Mario; (...); Nunez-Cortes, Jesus Millan

Article. 10.3390/v16030335. 2024

  • Open Access.

Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project.

Serrano-Gonzalo, Irene; (...); Giraldo, Pilar

Article. 10.1186/s13023-023-02939-4. 2023

  • Open Access.

Risk Categories in COVID-19 Based on Degrees of Inflammation: Data on More Than 17,000 Patients from the Spanish SEMI-COVID-19 Registry

Rubio-Rivas, M; (...); SEMI-COVID-19 Network

Article. 10.3390/jcm10102214. 2021

  • Open Access.

Severity Scores in COVID-19 Pneumonia: a Multicenter, Retrospective, Cohort Study.

Artero A; (...); Ramos-Rincón JM

Article. 10.1007/s11606-021-06626-7. 2021

  • Open Access.

The impact of frailty on intra-hospital survival in older patients with COVID-19 infection: the importance of early identification. SEMI-COVID National Registry.

Lopez-Sampalo, A; (...); Gomez-Huelgas, R

Article. 10.1016/j.rceng.2023.07.004. 2023


The role of attentional biases to appetitive stimuli in childhood overweight

Rojo-Bofill, LM; (...); Garcia-Blanco, A

Article. 10.1016/j.jecp.2019.04.014. 2019


The Use of Corticosteroids or Tocilizumab in COVID-19 Based on Inflammatory Markers.

Rubio-Rivas M; (...); Millán Núñez-Cortés J

Article. 10.1007/s11606-021-07146-0. 2021

  • Open Access.

Transferrin Isoforms, Old but New Biomarkers in Hereditary Fructose Intolerance

Cano, A; (...); de las Heras, J

Article. 10.3390/jcm10132932. 2021

  • Open Access.

Vitamin C and folate status in hereditary fructose intolerance

Cano, Ainara; (...); de las Heras, Javier

Article. 10.1038/s41430-022-01178-3. 2022

  • Open Access.

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