Risdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative Medicines

How We Think about Targeting RNA with Small Molecules

(2020) Matthew G. Costales et al.   JOURNAL OF MEDICINAL CHEMISTRY

Structural basis of a small molecule targeting RNA for a specific splicing correction

(2019) Sébastien Campagne et al.   Nature Chemical Biology

Molecular choreography of pre-mRNA splicing by the spliceosome

(2019) Ruixue Wan et al.   CURRENT OPINION IN STRUCTURAL BIOLOGY

Principles for targeting RNA with drug-like small molecules

(2018) Katherine Deigan Warner et al.   NATURE REVIEWS DRUG DISCOVERY

Mechanistic studies of a small-molecule modulator of SMN2 splicing

(2018) Jingxin Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)

(2018) Hasane Ratni et al.   JOURNAL OF MEDICINAL CHEMISTRY

Drugging the RNA World

(2018) Matthew D. Disney et al.   Cold Spring Harbor Perspectives in Biology

Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers

(2017) Manaswini Sivaramakrishnan et al.   Nature Communications

Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy

(2016) Hasane Ratni et al.   JOURNAL OF MEDICINAL CHEMISTRY

A comprehensive map of molecular drug targets

(2016) Rita Santos et al.   NATURE REVIEWS DRUG DISCOVERY

SMN2 splice modulators enhance U1–pre-mRNA association and rescue SMA mice

(2015) James Palacino et al.   Nature Chemical Biology

SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

(2014) N. A. Naryshkin et al.   SCIENCE