Molecular BiologyMolecular Therapy — Methods & Clinical DevelopmentPeer-reviewed
Patient-specific responses to SMN2 splice-modifying treatments in spinal muscular atrophy fibroblasts
Ilaria Signoria, Maria M Zwartkruis, Lotte Geerlofs…·Ewout J. N. Groen
The paper analyzes responses to SMN2 splice-modifying treatments in SMA fibroblasts, finding variability in treatment efficacy based on patient-specific factors.
10 citations