Latest Advances

  • Condition: Erythropoietic Protoporphyria (EPP)
  • Journal: Orphanet journal of rare diseases
  • Treatment Used: Afamelanotide
  • Number of Patients: 39
  • Published —
This study reported data on the effectiveness of afamelanotide treatment in erythropoietic protoporphyria (genetic skin disorder; EPP) and its phototoxic burn protection factor (PBPF).
  • Condition: Patients With Erythropoietic Protoporphyria (EPP)-Related Progressive Liver Disease
  • Journal: Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society
  • Treatment Used: Parenteral Protoporphyrin IX (PPIX)-Lowering Treatment
  • Number of Patients: 5
  • Published —
This study discusses the use of parenteral protoporphyrin IX (PPIX)-lowering therapy in the treatment of Patients With erythropoietic protoporphyria (an inherited metabolic disorder of heme synthesis resulting from overproduction of protoporphyrin; EPP)-Related Progressive Liver Disease.
  • Condition: Erythropoietic Protoporphyria (EPP)
  • Journal: Molecular genetics and metabolism
  • Treatment Used: Isoniazid (INH)
  • Number of Patients: 15
  • Published —
This study determined whether isoniazid (INH) might have therapeutic activity in patients with erythropoietic protoporphyria (EPP).
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