Found 4 Pompe Disease trials
A listing of Pompe Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
The purpose of this study is to assess the Safety and Efficacy of IONIS-FB-Lrx patients with Geographic Atrophy secondary to Age Related Macular Degeneration
This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: Study duration: Screening - up to 4 weeks; Primary Analysis Period (PAP) - 52 weeks; Extended Treatment Period …
A STUDY TO ASSESS THE LONG-TERM SAFETY AND EFFICACY OF INTRAVENOUS ATB200 CO-ADMINISTERED WITH ORAL AT2221 IN ADULT SUBJECTS WITH LATE-ONSET POMPE DISEASE
This study will test danicopan, a new drug that is being developed by Alexion to slow down progression of GA. The goals of this study are as follows: • To find out whether danicopan can slow the rate of GA disease progression, and in turn slow the loss of visual …
