Claudia A. Chiriboga

Claudia A. Chiriboga

Pediatric Neurology | Neuromusculoskeletal Medicine

180 Fort Washington Avenue
New York, NY 10032

Last Updated: 09/26/2026

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Found 35 publications

JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam.

JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam.

Journal: Journal of neurology

Published: January 26, 2024

Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.

Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.

Journal: Neurology and therapy

Published: July 03, 2023

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.

Journal: Neurology and therapy

Published: November 08, 2022

Pharmacotherapy for Spinal Muscular Atrophy in Babies and Children: A Review of Approved and Experimental Therapies.

Pharmacotherapy for Spinal Muscular Atrophy in Babies and Children: A Review of Approved and Experimental Therapies.

Journal: Paediatric drugs

Published: July 17, 2022

Digital measures of respiratory and upper limb function in spinal muscular atrophy: design, feasibility, reliability, and preliminary validity of a smartphone sensor-based assessment suite.

Digital measures of respiratory and upper limb function in spinal muscular atrophy: design, feasibility, reliability, and preliminary validity of a smartphone sensor-based assessment suite.

Journal: Neuromuscular disorders : NMD

Published: June 29, 2022

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.

Journal: Nature medicine

Published: February 18, 2022

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial.

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial.

Journal: Nature medicine

Published: February 18, 2022

Newborn Screening for Spinal Muscular Atrophy in New York State: Clinical Outcomes From the First 3 Years.

Newborn Screening for Spinal Muscular Atrophy in New York State: Clinical Outcomes From the First 3 Years.

Journal: Neurology

Published: February 03, 2022

Psychometric properties of the PEDI-CAT for children and youth with spinal muscular atrophy.

Psychometric properties of the PEDI-CAT for children and youth with spinal muscular atrophy.

Journal: Journal of pediatric rehabilitation medicine

Published: July 19, 2021

Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era.

Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era.

Journal: Journal of neuromuscular diseases

Published: March 01, 2021

Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study.

Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study.

Journal: The Lancet. Child & adolescent health

Published: January 18, 2021

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.

Journal: The Lancet. Neurology

Published: August 07, 2020

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Last Updated: 09/26/2026