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Risdiplam in Presymptomatic Spinal Muscular Atrophy

by Jong, Yuh-Jyh , Zanoteli, Edmar , Bertini, Enrico , Vlodavets, Dmitry , Gaki, Eleni , Wang, Yi , Mazurkiewicz-Bełdzińska, Maria , Fontoura, Paulo , Araujo, Alexandra P.Q.C. , Kletzl, Heidemarie , Jaber, Birgit , Wagner, Kathryn R. , Finkel, Richard S. , Gorni, Ksenija , Palfreeman, Laura , Rabbia, Michael , Nelson, Leslie , Servais, Laurent , Navas-Nazario, Aledie , Al-Muhaizea, Mohammad , Summers, Dave , Farrar, Michelle A.

in 76RS4S2ET1 (Risdiplam) / Action potential / Administration, Oral / Age / Asymptomatic Diseases - therapy / Azo Compounds / Babies / Body weight / Child, Preschool / Childhood Diseases / Clinical Medicine / Clinical Medicine General / Female / Gene Dosage / Genetic Testing / Genetics / Genetics General / Human health sciences / Humans / Infant / Infant, Newborn / Infants / Male / Medical treatment / mRNA / Neonatology / Neurologie / Neurology / Neuromuscular Agents / Neuromuscular Agents - administration & dosage / Neuromuscular Agents - adverse effects / Neuromuscular Agents/administration & dosage/adverse effects / Neuromuscular Disease / Neuroscience / Neurosurgery / Neurosurgery General / Nutritional Support - statistics & numerical data / Ostomy / Outpatient-Based Clinical Medicine / Patients / Pediatrics / Pediatrics General / Pharmacodynamics / Pharmacokinetics / Phenotypes / Proteins / Pyrimidines / Pédiatrie / Respiration, Artificial - statistics & numerical data / RNA Precursors / RNA Precursors - genetics / RNA Precursors - metabolism / RNA Precursors/genetics/metabolism / RNA Splicing - drug effects / RNA Therapeutics / Sciences de la santé humaine / SMN protein / SMN2 protein, human / Spinal Muscular Atrophies of Childhood - diagnosis / Spinal Muscular Atrophies of Childhood - drug therapy / Spinal Muscular Atrophies of Childhood - genetics / Spinal Muscular Atrophies of Childhood - mortality / Spinal Muscular Atrophies of Childhood/diagnosis/drug therapy/genetics/mortality / Spinal muscular atrophy / Survival / Survival of Motor Neuron 2 Protein / Survival of Motor Neuron 2 Protein - genetics / Treatment Outcome

2025

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Risdiplam in Presymptomatic Spinal Muscular Atrophy

2025

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Risdiplam in Presymptomatic Spinal Muscular Atrophy

2025

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Journal Article

Risdiplam in Presymptomatic Spinal Muscular Atrophy

Jong, Yuh-Jyh,

Zanoteli, Edmar,

Bertini, Enrico,

Vlodavets, Dmitry,

Gaki, Eleni,

Wang, Yi,

Mazurkiewicz-Bełdzińska, Maria,

Fontoura, Paulo,

Araujo, Alexandra P.Q.C.,

Kletzl, Heidemarie,

Jaber, Birgit,

Wagner, Kathryn R.,

Finkel, Richard S.,

Gorni, Ksenija,

Palfreeman, Laura,

Rabbia, Michael,

Nelson, Leslie,

Servais, Laurent,

Navas-Nazario, Aledie,

Al-Muhaizea, Mohammad,

Summers, Dave,

Farrar, Michelle A.

2025

Overview

In infants with genetically diagnosed spinal muscular atrophy, risdiplam therapy before the development of symptoms led to improved motor milestones and survival at 12 and 24 months. Treatment-related adverse events were mild.

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Publisher

Massachusetts Medical Society

Subject

76RS4S2ET1 (Risdiplam)

/ Action potential

/ Administration, Oral

/ Age

/ Asymptomatic Diseases - therapy

/ Azo Compounds

/ Babies