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A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia.
Fields, T; Patterson, M; Bremova-Ertl, T; Belcher, G; Billington, I; Churchill, G C; Davis, W; Evans, W; Flint, S; Galione, A; Granzer, U; Greenfield, J; Karl, R; Kay, R; Lewi, D; Mathieson, T; Meyer, T; Pangonis, D; Platt, F M; Tsang, L; Verburg, C; Factor, M; Strupp, M.
Afiliação
  • Fields T; IntraBio Ltd, Begbroke Science Park, Begbroke Hill, Woodstock Road, Oxford, OX5 1PF, UK. tfields@intrabio.com.
  • Patterson M; Mayo Clinic, 200 First Street SW, Rochester, MN, 55905, USA.
  • Bremova-Ertl T; Department of Neurology, Inselspital, University Hospital Bern and University of Bern, Bern, Switzerland.
  • Belcher G; PV Consultancy, 113 St Georges Square Mews, London, SW1V 3RZ, UK.
  • Billington I; IntraBio Ltd, Begbroke Science Park, Begbroke Hill, Woodstock Road, Oxford, OX5 1PF, UK.
  • Churchill GC; Department of Pharmacology, University of Oxford, Mansfield Road, Oxford, OX1 3QT, UK.
  • Davis W; Ataxia-Telangiectasia Society, Rothamsted Experimental Station West Common, Harpenden, AL5 2JQ, UK.
  • Evans W; Niemann-Pick UK, Vermont House, Concord, Washington, Tyne and Wear, NE37 2SQ, UK.
  • Flint S; Primary Care Stratified Medicine (PRISM) Division of Primary Care, University of Nottingham, Nottingham, UK.
  • Galione A; IntraBio Ltd, Begbroke Science Park, Begbroke Hill, Woodstock Road, Oxford, OX5 1PF, UK.
  • Granzer U; Department of Pharmacology, University of Oxford, Mansfield Road, Oxford, OX1 3QT, UK.
  • Greenfield J; Granzer Regulatory Consulting & Services, Kistlerhofstr. 172C, D-81379, Munich, Germany.
  • Karl R; Ataxia UK, 12 Broadbent close, London, N6 5JW, UK.
  • Kay R; Cure Tay-Sachs Foundation, 2409 E. Luke Avenue, Phoenix, AZ, 85016, USA.
  • Lewi D; RK Statistics, Brook House, Mesne Lane, Bakewell, DE45 1AL, UK.
  • Mathieson T; The Cure & Action for Tay-Sachs Foundation, 94 Milborough Crescent, Lee, London, SE12 0RW, UK.
  • Meyer T; International Niemann-Pick Disease Alliance, Vermont House, Concord, Washington, Tyne and Wear, NE37 2SQ, UK.
  • Pangonis D; Granzer Regulatory Consulting & Services, Kistlerhofstr. 172C, D-81379, Munich, Germany.
  • Platt FM; National Tay-Sachs and Allied Disease Foundation, 2001 Beacon Street, Suite 204, Boston, MA, 02135, USA.
  • Tsang L; Department of Pharmacology, University of Oxford, Mansfield Road, Oxford, OX1 3QT, UK.
  • Verburg C; Arnold & Porter Kaye Scholer LLP, 25 Old Broad Street, London, EC2N 1HQ, UK.
  • Factor M; IntraBio Ltd, Begbroke Science Park, Begbroke Hill, Woodstock Road, Oxford, OX5 1PF, UK.
  • Strupp M; IntraBio Ltd, Begbroke Science Park, Begbroke Hill, Woodstock Road, Oxford, OX5 1PF, UK.
Trials ; 22(1): 84, 2021 Jan 22.
Article em En | MEDLINE | ID: mdl-33482890
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Ataxia Telangiectasia / Doenças Neurodegenerativas / Gangliosidoses GM2 Tipo de estudo: Diagnostic_studies / Guideline Limite: Female / Humans / Male Idioma: En Revista: Trials Assunto da revista: MEDICINA / TERAPEUTICA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Reino Unido
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Ataxia Telangiectasia / Doenças Neurodegenerativas / Gangliosidoses GM2 Tipo de estudo: Diagnostic_studies / Guideline Limite: Female / Humans / Male Idioma: En Revista: Trials Assunto da revista: MEDICINA / TERAPEUTICA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Reino Unido