6 results
To evaluate the safety and efficacy of PRX-102 in patients with Fabry disease currently treated with agalsidase alfa
primaryTo assess the safety and tolerability of multiple, once-daily oral doses of RDN-929 over 28 days in healthy older adult subjects and early symptomatic AD subjects.secondaryTo assess the plasma and CSF pharmacokinetics (PK) of RDN-929 in…
To evaluate the safety and efficacy of PRX-102 compared to agalsidase beta in Fabry disease patients with impaired renal function.
Therefore we aimed to study the 24 hour effect of twice daily aclidinium/formeterol versus once daily tiotropium on serial static and dynamic lung hyperinflation measurements, as well as (24 hour) symptoms and sleep quality. Tiotropium was chosen as…
Primary objectives: to evaluate the efficacy of three different single doses of PHA-022121 versus placebo in achieving angioedema symptom relief during acute attacks and the efficacy of prophylactic treatment with PHA-022121 versus placebo in…
To evaluate the ongoing safety, tolerability, and efficacy parameters of pegunigalsidase alfa in adult Fabry patients who have successfully completed studies PB-102-F20 and PB-102-F30, or completed at least 48 months in study PB-102-F03.