Real-Life Effectiveness After Switching to Avalglucosidase Alfa in Late-Onset Pompe Disease Patients Worsening on Alglucosidase Alfa Therapy: A French Cohort Study.
Patients with a progressive muscle disease who switched to a newer enzyme therapy experienced disease stabilization and improved function.
Late-onset Pompe disease (LOPD) is a progressive myopathy caused by acid alpha-glucosidase deficiency, leading to glycogen accumulation in skeletal and cardiac muscle. For patients experiencing significant walking decline under alglucosidase alfa therapy, switching to avalglucosidase alfa resulted in disease stabilization, with mild improvement in the first year and a return to pre-switch baseline functional capacity thereafter.
What the study was
- Study design
- Phase 3 clinical trial
- Population
- Cancer/disease patients
- Category
- Drug Development
- Maturity
- Potentially Practice-Changing
- Journal
- European journal of neurology
Why it surfaced
Matched topic(s): Rare diseases with high unmet need. Study design: Phase 3 clinical trial. Score 7/10.
A plain-language summary of published research — not medical advice. Talk to a clinician about your own care.