4 results
Approved WMOCompleted
The aim is to assess the long-term safety of topical use of ZORBLISA in patients with Epidermolysis Bullosa (EB).
Approved WMOCompleted
The primary objective is to compare the efficacy and safety of SD-101-6.0vs. SD- 101-0.0 (placebo) in patients with Simplex, RecessiveDystrophic, or Junctional non Herlitz Epidermolysis Bullosa.The primary endpoint is the complete closure of the…
Approved WMOCompleted
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
Approved WMORecruiting
• To evaluate the efficacy of VX-147 to reduce proteinuria• To evaluate the efficacy of VX 147 on renal function as measured by eGFR slope