Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls | Zendy

Basil T. Darras | Zendy; Riccardo Masson | Zendy; Maria Mazurkiewicz-Bełdzińska | Zendy; Kristy Rose | Zendy; Hui Xiong | Zendy; Edmar Zanoteli | Zendy; Giovanni Baranello | Zendy; Claudio Bruno | Zendy; D. Vlodavets | Zendy; Yi Wang | Zendy; Muna El-Khairi | Zendy; Marianne Gerber | Zendy; Ksenija Gorni | Zendy; Omar Khwaja | Zendy; H. Kletzl | Zendy; R. Scalco | Zendy; Paulo Fontoura | Zendy; Laurent Servais | Zendy

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Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls

Author(s) -

Basil T. Darras,

Riccardo Masson,

Maria Mazurkiewicz-Bełdzińska,

Kristy Rose,

Hui Xiong,

Edmar Zanoteli,

Giovanni Baranello,

Claudio Bruno,

D. Vlodavets,

Yi Wang,

Muna El-Khairi,

Marianne Gerber,

Ksenija Gorni,

Omar Khwaja,

H. Kletzl,

R. Scalco,

Paulo Fontoura,

Laurent Servais

Publication year - 2021

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa2102047

Subject(s) - sma* , spinal muscular atrophy , medicine , natural history , smn1 , pediatrics , neuromuscular disease , milestone , physical medicine and rehabilitation , disease , mathematics , combinatorics , archaeology , history

Type 1 spinal muscular atrophy (SMA) is a progressive neuromuscular disease characterized by an onset at 6 months of age or younger, an inability to sit without support, and deficient levels of survival of motor neuron (SMN) protein. Risdiplam is an orally administered small molecule that modifies SMN2 pre-messenger RNA splicing and increases levels of functional SMN protein in blood.

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