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: FIREFISH Part 2: Efficacy and safety of risdiplam (RG7916 in infants with Type 1 spinal muscular atrophy (SMA

Researchers

Presenter

  • Giovanni Baranello

Principal Investigators

  • Laurent Servais

  • Riccardo Masson

  • Maria Mazurkiewicz-Bełdzińska

  • Kristy Rose

  • Dmitry Vlodavets

  • Hui Xiong

  • Edmar Zanoteli

  • Muna El-Khairi

  • Sabine Fuerst-Recktenwald

  • Marianne Gerber

  • Ksenija Gorni

  • Heidemarie Kletzl

  • Renata Scalco

  • Basil T. Darras

Medical Centers

  • Fondazione IRCCS Istituto Neurologico Besta, Developmental Neurology Unit - Milan (Italy; The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, United Kingdom

  • Reference Center for Neuromuscular Disease, Centre Hospitalier Régional de La Citadelle - Liège (Belgium; MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, United Kingdom

  • Fondazione IRCCS Istituto Neurologico Besta, Developmental Neurology Unit, Milan, Italy

  • Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland

  • Paediatric Gait Analysis Service of New South Wales, The Children’s Hospital at Westmead, Sydney, Australia

  • Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute of Pirogov Russian National Research Medical University

  • Department of General Surgery , Peking University First Hospital, Beijing, China

  • Department of Neurology, Hospital das Clínicas, University of São Paulo, São Paulo, Brazil

  • Roche Products Ltd., Welwyn Garden City, United Kingdom

  • Pharma Development Oncology, F. Hoffmann-La Roche, Basel, Switzerland

  • Pharma Development Oncology, F. Hoffmann-La Roche, Basel, Switzerland

  • PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland

  • Roche Pharma Research and Early Development (pRED), Roche Innovation Center Basel, Basel, Switzerland

  • Boston Children’s Hospital, Harvard Medical School, Boston, MA, United States of America

Locations

  • United Kingdom

  • Italy

  • Poland

  • Australia

  • Russia

  • China

  • Brazil

  • Switzerland

  • United States

Companies

  • Feng Chi Biotech Cor

Study Components

Therapeutic Area

  • Central Nervous System (CNS)

  • Genetic Disorder

Disease

  • Spinal muscular atrophy

Biomarkers

  • N/A

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • Pharmacokinetics

  • Pharmacodynamics

  • Open Label

  • Multicenteric

Phase

  • NA

Study Id's

  • NCT02913482

Sponsors

  • N/A

Result

  • N/A

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