Birgit Jaber
Basel, BS, Switzerland
Last Updated: 09/26/2026
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Found 8 publications
Risdiplam in Presymptomatic Spinal Muscular Atrophy.
Journal: The New England journal of medicine
Published: August 13, 2025
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.
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.
Journal: Neurology and therapy
Published: November 08, 2022
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial.
Journal: The Lancet. Neurology
Published: April 08, 2022
Effect of mild or moderate hepatic impairment on the pharmacokinetics of risdiplam.
Journal: British journal of clinical pharmacology
Published: September 29, 2021
Risdiplam treatment has not led to retinal toxicity in patients with spinal muscular atrophy.
Journal: Annals of clinical and translational neurology
Published: July 23, 2020
A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier.
Journal: British journal of clinical pharmacology
Published: March 23, 2018
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Last Updated: 09/26/2026