Experiences from treating seven adult 5q spinal muscular atrophy patients with Nusinersen.

Elisabeth JochmannRobert SteinbachThomas JochmannHa-Yeun ChungAnnekathrin RödigerRotraud NeumannThomas E MayerKlaus KirchhofDana Loudovici-KrugUlrich C SmolenskiOtto W WitteJulian Grosskreutz

Published in: Therapeutic advances in neurological disorders (2020)

Treating adult SMA patients with Nusinersen is feasible and most patients consider it beneficial. It demands a complex organizational and interdisciplinary effort. Due to the slowly decreasing motor functions in adult SMA patients, long observation phases for this recently approved treatment are needed to allow conclusions about effectiveness of Nusinersen in adults.

Keyphrases

end stage renal disease
newly diagnosed
ejection fraction
systematic review
prognostic factors
randomized controlled trial
peritoneal dialysis
mental health