Risdiplam in Type 1 Spinal Muscular Atrophy.

Giovanni Baranello,Basil T. Darras,John W. Day,Nicolas Deconinck,Andrea Klein,Riccardo Masson,Eugenio Mercuri,Kristy J. Rose,Muna El-Khairi,Marianne Gerber,Ksenija Gorni,Omar Khwaja,Heidemarie Kletzl,Renata S. Scalco,Timothy Seabrook,Paulo Fontoura,Laurent Servais

Risdiplam in Type 1 Spinal Muscular Atrophy.

2021

Giovanni Baranello
Basil T. Darras
John W. Day
Nicolas Deconinck
Andrea Klein
Riccardo Masson
Eugenio Mercuri
Kristy J. Rose
Muna El-Khairi
Marianne Gerber
Ksenija Gorni
Omar Khwaja
Heidemarie Kletzl
Renata S. Scalco
Timothy Seabrook
Paulo Fontoura
Laurent Servais

Abstract Background Type 1 spinal muscular atrophy is a rare, progressive neuromuscular disease that is caused by low levels of functional survival of motor neuron (SMN) protein. Risdiplam is an or...

Keywords:

Pathology
Neuromuscular disease
Spinal muscular atrophy
Medicine
Survival of motor neuron

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