Lutz O. Muller
Basel, BS, Switzerland
Last Updated: 09/26/2026
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 24 publications
Risdiplam: A Small Molecule mRNA Splice Modifier Approved to Treat SMA Disease.
Journal: Toxicologic pathology
Published: November 18, 2025
Further Comments on Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy. Reply.
Journal: The New England journal of medicine
Published: July 02, 2025
Developmental and Reproductive Toxicity Testing Strategies for Oligonucleotides: A Workshop Proceedings.
Journal: Nucleic acid therapeutics
Published: May 30, 2025
Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy.
Journal: The New England journal of medicine
Published: February 19, 2025
Angelman syndrome patient-derived neuron screen leads to clinical ASO rugonersen targeting UBE3A-ATS with long-lasting effect in monkeys.
Journal: Nucleic acids research
Published: February 10, 2025
Male Reproduction in Spinal Muscular Atrophy (SMA) and the Potential Impact of Oral Survival of Motor Neuron 2 (SMN2) Pre-mRNA Splicing Modifiers.
Journal: Neurology and therapy
Published: March 05, 2024
Discovery of Orally Available and Brain Penetrant AEP Inhibitors.
Journal: Journal of medicinal chemistry
Published: December 13, 2023
Addressing Today's Absorption, Distribution, Metabolism, and Excretion (ADME) Challenges in the Translation of In Vitro ADME Characteristics to Humans: A Case Study of the SMN2 mRNA Splicing Modifier Risdiplam.
Journal: Drug metabolism and disposition: the biological fate of chemicals
Published: June 03, 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
Rewriting the (tran)script: Application to spinal muscular atrophy.
Journal: Progress in medicinal chemistry
Published: March 19, 2019
Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs.
Journal: Pharmacology research & perspectives
Published: October 10, 2018
Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA).
Journal: Journal of medicinal chemistry
Published: July 26, 2018
Last Updated: 09/26/2026