CONTEXT: Long term treatment effect study of a rare disease (LOPD) | Relatively low patient numbers for RWR, but the findings just as powerful for this rare disease.
IMPACT: medium
READ TIME: 5 mins
Quality Level Mean [1 – 10]: 8
1. “In the first years of treatment, Myozyme (alglucosidase alfa) — marketed in the U.S. as Lumizyme — can effectively increase physical endurance, and improve lung function and muscle strength in adults with late-onset Pompe disease (LOPD), a real-world study found.”
2. “Yet, despite this treatment, patients’ clinical outcomes, particularly lung function, tend to deteriorate over time, indicating that additional efforts must be made to improve Myozyme’s effectiveness.”
3. “Several studies have reported that Myozyme can significantly improve patients’ clinical outcomes in the first three to five years of treatment.”
4. “Muscle strength, lung function, and walking abilities were evaluated at the study’s start (baseline) and then once each year during follow-up.”
5. “Of all the outcome measures evaluated, walking abilities and physical endurance — assessed by the six-minute walk test (6MWT) — were the ones that worsened the least over time, with most patients still experiencing positive effects from the treatment after seven years of follow-up.”