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Rimeporide as a first- in-class NHE-1 inhibitor: Results of a phase Ib trial in young patients with Duchenne Muscular Dystrophy.
Previtali, Stefano C; Gidaro, Teresa; Díaz-Manera, Jordi; Zambon, Alberto; Carnesecchi, Stephanie; Roux-Lombard, Pascale; Spitali, Pietro; Signorelli, Mirko; Szigyarto, Cristina Al-Khalili; Johansson, Camilla; Gray, Julian; Labolle, Delphine; Porte Thomé, Florence; Pitchforth, Jacqueline; Domingos, Joana; Muntoni, Francesco.
Afiliação
  • Previtali SC; IRCCS San Raffaele Scientific Institute, Department of Neurology and INSPE, Milan, Italy.
  • Gidaro T; Institute of Myology, Hopital Trousseau, I- Motion, Paris, France.
  • Díaz-Manera J; Hospital de la Santa Creu i Sant Pau de Barcelona Servei de Neurologia, Barcelona, Spain; Centro de Investigación Biomédica en Red en Enfermedades Raras (CIBERER), Spain.
  • Zambon A; IRCCS San Raffaele Scientific Institute, Department of Neurology and INSPE, Milan, Italy.
  • Carnesecchi S; Geneva University (UNIGE), Geneva, Switzerland.
  • Roux-Lombard P; Geneva University Hospital (HUG), Immunology and Allergology Department, Geneva, Switzerland.
  • Spitali P; Leiden University Medical Center, Netherlands.
  • Signorelli M; Leiden University Medical Center, Netherlands.
  • Szigyarto CA; KTH, School of Chemistry, Biotechnology and Health, Royal Institute of Technology, Stockolm, Sweden.
  • Johansson C; Science for Life Laboratory, Department of Protein Science, Division of Systems Biology, Solna, Sweden.
  • Gray J; EspeRare, Geneva, Switzerland.
  • Labolle D; EspeRare, Geneva, Switzerland.
  • Porte Thomé F; EspeRare, Geneva, Switzerland. Electronic address: porte.florence@esperare.org.
  • Pitchforth J; UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital Dubowitz Neuromuscular Centre, London, UK.
  • Domingos J; UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital Dubowitz Neuromuscular Centre, London, UK.
  • Muntoni F; UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital Dubowitz Neuromuscular Centre, London, UK; NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, Great Ormond Street Hospital Trust, University College London,
Pharmacol Res ; 159: 104999, 2020 09.
Article em En | MEDLINE | ID: mdl-32535224
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Músculo Esquelético / Distrofia Muscular de Duchenne / Trocador 1 de Sódio-Hidrogênio / Fármacos Neuromusculares Tipo de estudo: Clinical_trials / Diagnostic_studies / Prognostic_studies Limite: Child / Humans / Male País/Região como assunto: Europa Idioma: En Revista: Pharmacol Res Ano de publicação: 2020 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Músculo Esquelético / Distrofia Muscular de Duchenne / Trocador 1 de Sódio-Hidrogênio / Fármacos Neuromusculares Tipo de estudo: Clinical_trials / Diagnostic_studies / Prognostic_studies Limite: Child / Humans / Male País/Região como assunto: Europa Idioma: En Revista: Pharmacol Res Ano de publicação: 2020 Tipo de documento: Article