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Protoporphyria, Erythropoietic
An autosomal dominant porphyria that is due to a deficiency of FERROCHELATASE (heme synthetase) in both the LIVER and the BONE MARROW, the last enzyme in the 8-enzyme biosynthetic pathway of HEME. Clinical features include mainly neurological symptoms, rarely cutaneous lesions, and elevated levels of protoporphyrin and COPROPORPHYRINS in the feces.
Drugs by status
Approved for this indication (2)
Phase 3 trials (4)
Phase 2 trials (2)
Organizations
Organization Involved with Phase 3 Indications (11)
- Clinuvel Pharmaceuticals Limited
- Maastricht University
- Harvard University
- University of Manchester
- University of Toronto
- Royal College of Surgeons, Ireland
- Mitsubishi Tanabe
- St Woolos Hospital, Newport
- Centre Francais des Porphyries, Hopital Louis Mourier, Colombes, France
- Erasmus University Rotterdam
- University of Helsinki
Organization Involved with Phase 2 Indications (1)
Organization Involved with Other Experimental Indications (7)