What was studied?
Ten participants entered the open-label extension of TAZPOWER and eight reached the 168-week visit. Researchers assessed tolerability, walking performance, fatigue, strength, cardiac measurements and cardiolipin-related biomarkers.
What did the researchers report?
Functional and fatigue measurements improved relative to extension baseline, and some cardiac measures also changed. Injection-site reactions were the most commonly reported adverse events. The publication adds information about longer observation in a rare disease.
What are the limits?
This is a follow-up of the TAZPOWER cohort, not a separate placebo-controlled efficacy trial. Survivor and participation effects, small numbers and lack of a concurrent control limit causal interpretation. Long-term changes do not establish safety or benefit for unrelated populations or catalog materials.
Original editorial summary by Sulan Peptide. Based on the source abstract; consult the full paper for methods, analyses, funding and disclosures.