Summarized by Masters of Longevity from PR Newswire.
Long-term clinical data on a combination therapy for late-onset Pompe disease, highlighting what readers can expect from the research-focused article.
Key Takeaways
- Eighty-two participants received continuous combination therapy from the original trial baseline through five years of follow-up.
- Motor function outcomes were durable and pulmonary function showed relative stabilization over the five-year observation period.
- The open-label extension included both prior ERT-experienced patients and ERT-naive patients, with prior ERT duration averaging 7.6 years for experienced individuals.
