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Transthyretin stabilization activity of the catechol-O-methyltransferase inhibitor tolcapone (SOM0226) in hereditary ATTR amyloidosis patients and asymptomatic carriers: proof-of-concept study.
Gamez, Josep; Salvadó, María; Reig, Núria; Suñé, Pilar; Casasnovas, Carles; Rojas-Garcia, Ricard; Insa, Raúl.
Affiliation
  • Gamez J; a Neuromuscular Disorders Clinic, Department of Neurology , Vall d'Hebron University Hospital, VHIR, European Reference Network on Rare, Neuromuscular Disorders (ERN EURO-NMD), UAB , Barcelona , Spain.
  • Salvadó M; a Neuromuscular Disorders Clinic, Department of Neurology , Vall d'Hebron University Hospital, VHIR, European Reference Network on Rare, Neuromuscular Disorders (ERN EURO-NMD), UAB , Barcelona , Spain.
  • Reig N; b Research and Development Department , SOM Biotech, S.L , Barcelona , Spain.
  • Suñé P; c Pharmacy Department, Vall d'Hebron Research Institute (VHIR) , Hospital Universitari Vall d'Hebron , Barcelona , Spain.
  • Casasnovas C; d Neuromuscular Disorders Unit, Neurology Department , Bellvitge University Hospital - IDIBELL , Barcelona , Spain.
  • Rojas-Garcia R; e Department of Neurology, Neuromuscular Diseases Unit Hospital de la Santa Creu i Sant Pau , Center for Networked Biomedical Research into Rare Diseases (CIBERER), UAB , Barcelona , Spain.
  • Insa R; b Research and Development Department , SOM Biotech, S.L , Barcelona , Spain.
Amyloid ; 26(2): 74-84, 2019 Jun.
Article in En | MEDLINE | ID: mdl-31119947
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Prealbumin / Amyloid Neuropathies, Familial / Protein Aggregation, Pathological / Proof of Concept Study / Tolcapone Type of study: Prognostic_studies Limits: Adult / Aged / Female / Humans / Male / Middle aged Language: En Journal: Amyloid Journal subject: BIOQUIMICA Year: 2019 Document type: Article Affiliation country: Spain Country of publication: United kingdom
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Prealbumin / Amyloid Neuropathies, Familial / Protein Aggregation, Pathological / Proof of Concept Study / Tolcapone Type of study: Prognostic_studies Limits: Adult / Aged / Female / Humans / Male / Middle aged Language: En Journal: Amyloid Journal subject: BIOQUIMICA Year: 2019 Document type: Article Affiliation country: Spain Country of publication: United kingdom