A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment.

ABSTRACT:

Background

Studies 4658-201/202 (201/202) evaluated treatment effects of eteplirsen over 4 years in patients with Duchenne muscular dystrophy and confirmed exon-51 amenable genetic mutations. Chart review Study 4658-405 (405) further followed these patients while receiving eteplirsen during usual clinical care.

Objective

To compare long-term clinical outcomes of eteplirsen-treated patients from Studies 201/202/405 with those of external controls.

Methods

Median total follow-up time was approximately 6 years of eteplirsen treatment. Outcomes included loss of ambulation (LOA) and percent-predicted forced vital capacity (FVC%p). Time to LOA was compared between eteplirsen-treated patients and standard of care (SOC) external controls and was measured from eteplirsen initi

SUBMITTER: Mitelman O 

PROVIDER: S-EPMC8842766 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature