REPLACE Registry
Investigational therapy: Cholic acid
Therapeutic area: BASD; PBD-ZSD with liver involvement
Study type: Observational
Learn more: NCT03115086
ACAPPELLA Study
Natural History Study of Homocystinuria Caused by Cystathionine Beta-synthase Deficiency
Therapeutic area: HCU
Study type: Observational
Learn more: hcuconnection.com | NCT02998710
EPPIK Study
Sparsentan Treatment in Pediatrics with Proteinuric Glomerular Diseases (EPPIK)
Therapeutic area: FSGS; IgAN
Study type: Phase 2
Learn more: eppikclinicalstudy.com | NCT05003986
RESTORE Study
Investigational therapy: Chenodeoxycholic acid
Therapeutic area: CTX
Study type: Phase 3
Learn more: CTXrestore.com | NCT04270682
COMPOSE Study
Investigational therapy: Pegtibatinase (TVT-058)
Therapeutic area: HCU
Study type: Phase 1/2
Learn more: hcuconnection.com | NCT03406611
HONUS Study
Humanistic Burden of FSGS and IgAN
Therapeutic area: FSGS; IgAN
Study type: Observational
Learn more: NCT05200871
