Pipeline

Product/Program Pre-Clinical Phase I Phase II Phase III Market
Cholbam:
Bile acid synthesis disorders due to single enzyme defects
Peroxisomal disorders
Thiola:
Cystinuria
Chenodal:
Gallstones/CTX*
Sparsentan:
Focal Segmental Glomerulosclerosis  
RE-024:
Pantothenate Kinase Associated Neurodegeneration  
RE-034:
Multiple Indications      
Discovery Programs:
Undisclosed Rare Disease Targets      

* Chenodal is not currently indicated for CTX but is the standard of care for that disease.