Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.

Claudia A ChiribogaClaudio BrunoTina DuongDirk FischerEugenio MercuriJanbernd KirschnerAnna Kostera-PruszczykBirgit JaberKsenija GorniHeidemarie KletzlImogen CarruthersCarmen MartinFrancis WarrenRenata S ScalcoKathryn R WagnerFrancesco Muntoninull null

Published in: Neurology and therapy (2023)

The safety and PD of risdiplam in patients who were previously treated were consistent with those of treatment-naïve patients.

Keyphrases

newly diagnosed
end stage renal disease
ejection fraction
prognostic factors
peritoneal dialysis