3 results
Approved WMOCompleted
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
Approved WMOCompleted
The objective of this pivotal study is to assess the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of RP103 in pediatric and adult patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support…
Approved WMOCompleted
The primary objectives of this study are: • To determine the longterm safety and tolerability of rhHNS administered via an intrathecal (IT) route once monthly for 8 years in patients with MPS IIIA, who have received and tolerated 6 months of…