© 2020 F. Hoffmann-La Roche Ltd. All rights reserved.
FIREFISH Part 2: Efficacy and safety of risdiplam (RG7916) in infants
with Type 1 spinal muscular atrophy (SMA)
Laurent Servais,
1–3*
Giovanni Baranello,
4,5
Riccardo Masson,
4
Maria Mazurkiewicz-Bełdzińska,
6
Kristy
Rose,
7
Dmitry Vlodavets,
8
Hui Xiong,
9
Edmar Zanoteli,
10
Muna El-Khairi,
11
Sabine Fuerst-Recktenwald,
12
Marianne Gerber,
13
Ksenija Gorni,
14
Heidemarie Kletzl,
15
Renata Scalco,
12
Basil T. Darras
16
on behalf of
the FIREFISH Working Group
1
Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liège & University of Liège,
Belgium;
2
MDUK Oxford Neuromuscular Center, Department of Paediatrics, University of Oxford, Oxford, UK;
3
I-Motion - Hôpital Armand Trousseau, Paris,
France;
4
Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy;
5
The Dubowitz Neuromuscular Centre, NIHR Great
Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, & Great Ormond Street
Hospital Trust , London, UK;(current);
6
Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland;
7
Paediatric Gait Analysis
Service of New South Wales, The Children’s Hospital at Westmead, Sydney, Australia;
8
Russian Children Neuromuscular Center, Veltischev Clinical Pediatric
Research Institute of Pirogov Russian National Research Medical University, Moscow, Russia;
9
Department of Pediatrics, Peking University First Hospital,
Beijing, China;
10
Department of Neurology, Hospital das Clínicas, University of São Paulo, São Paulo, Brazil;
11
Roche Products Ltd., Welwyn Garden City, UK;
12
Pharma Development Neurology, F. Hoffmann-La Roche Ltd., Basel, Switzerland;
13
Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel,
Switzerland;
14
PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd., Basel, Switzerland;
15
Roche Pharmaceutical Research and Early
Development, Roche Innovation Center Basel, Basel, Switzerland;
16
Boston Children’s Hospital, Harvard Medical School, Boston, MA, USA.
*Presenter