P. Vazirani

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

F. Metzger, E. Pinard, W. Muster, 2016, Journal of medicinal chemistry.

P3.12 Small molecule compounds that correct alternative splicing of the SMN2 gene and restore SMN protein expression and function

S. Peltz, D. Kobayashi, S. Paushkin, 2011, Neuromuscular Disorders.

The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential

S. Peltz, Jairo L. Sierra, H. Ren, 2018, PloS one.

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

Anirvan Ghosh, Roland Schmucki, Hasane Ratni, 2014, Science.