Work

FIREFISH Part 2: 24-month Efficacy and Safety of Risdiplam in Infants with Type 1 Spinal Muscular Atrophy (SMA) (4126)

Year: 2021

Type: article

Source: Neurology

DOI: 10.1212/wnl.96.15_supplement.4126

Authors: Basil T. Darras, Ricardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose, Hui Xiong, Edmar Zanoteli, Giovanni Baranello, Dmitry Vlodavets, Angela Dodman, Muna El-Khairi, Marianne Gerber, Ksenija Gorni, Heidemarie Kletzl, Renata S. Scalco, Laurent Servais

Institutions: Boston Children's Hospital, Harvard University, Fondazione IRCCS Istituto Neurologico Carlo Besta, Gdańsk Medical University, Children's Hospital at Westmead, Westmead Hospital, Peking University First Hospital, Universidade de São Paulo, NIHR Great Ormond Street Hospital Biomedical Research Centre, University College London, Pirogov Russian National Research Medical University, F. Hoffmann-La Roche AG, University of Liège, University of Oxford, Hôpital Armand-Trousseau, Centre Hospitalier Universitaire de Liège

Language: English

Topic: Neurogenetic and Muscular Disorders Research

Subfield: Genetics (clinical)

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W3206032012

FWCI: 0.807

Cites:

Cited by: 4

Related to: 10