Type 1 spinal muscular atrophy treated with nusinersen in Norway, a five-year follow-up [PDF]
Background: New treatments for 5q spinal muscular atrophy (SMA) have led to changes in the disease phenotype. Questions about long-term efficacy, however, persist.
Hagen, Milada +7 more
core +1 more source
Brain and BehaviorObjective This study was based on a retrospective clinical observational cohort study of a two‐center application of nusinersen in China to evaluate the clinical efficacy and adverse effects of nusinersen in the treatment of SMA (spinal muscular atrophy)
Dan Li +5 more
doaj +1 more source
Effective local treatment of necrotizing fasciitis using a chlorine solution obtained by electrolysis [PDF]
Srpski Arhiv za Celokupno LekarstvoIntroduction. Spinal muscular atrophy (SMA) and acute lymphoblastic leukemia (ALL) are rare diseases, with usual onset in childhood. To date, no cases have been reported where these conditions co-exist in one patient.
Kovačević Predrag T.
doaj +1 more source
Priority setting at the clinical level: the case of nusinersen and the Norwegian national expert group [PDF]
, 2021 Morten Magelssen +3 more
openalex +1 more source
Pharmacology Research &Perspectives, Volume 13, Issue 4, August 2025.
Ida Štimac +3 more
wiley +1 more source
Nusinersen in type 0 spinal muscular atrophy: should we treat? [PDF]
, 2020 Eloisa Tiberi +9 more
openalex +1 more source
Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy [PDF]
, 2018 Kevin A. Strauss +10 more
openalex +1 more source
Correction to: Nusinersen: A Review in 5q Spinal Muscular Atrophy [PDF]
, 2021 Sheridan M. Hoy
openalex +1 more source
Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps [PDF]
, 2018 Teresa Gidaro, Laurent Servais
openalex +1 more source
Short review on determination of nusinersen for spinal muscular atrophy treatment [PDF]
Preprint artykułuNusinersen, an antisense oligonucleotide, is an active ingredient of the first drug approved by the Food and Drug Administration for the treatment of spinal muscular atrophy, a genetic condition characterized by progressive muscle ...
Balińska, Natalia +2 more
core +1 more source