Serum creatine kinase and creatinine in adult spinal muscular atrophy under nusinersen treatment.

ABSTRACT:

Objective

To determine whether serum creatine kinase activity (CK) and serum creatinine concentration (Crn) are prognostic and predictive biomarkers for disease severity, disease progression, and nusinersen treatment effects in adult patients with 5q-associated spinal muscular atrophy (SMA).

Methods

Within this retrospective, multicenter observational study in 206 adult patients with SMA, we determined clinical subtypes (SMA types, ambulatory ability) and repeatedly measured CK and Crn and examined disease severity scores (Hammersmith Functional Motor Scale Expanded, Revised Upper Limb Module, and revised Amyotrophic Lateral Sclerosis Functional Rating Scale). Patients were followed under nusinersen treatment for 18 months.

Results

CK and Crn differed between clinical

SUBMITTER: Freigang M 

PROVIDER: S-EPMC8108420 | biostudies-literature | 2021 May

REPOSITORIES: biostudies-literature