THE PURPOSE OF THIS GRANT IS TO SELECT APPROVABLE DRUGS FOR THE TREATMENT OF FOUR ORPHAN DISEASES: SICKLE CELL DISEASE,THALASSEMIA, HEMOCHROMATOSIS, AND ERYTHROPOIETIC PORPHYRIAS.

THE PURPOSE OF THIS GRANT IS TO SELECT APPROVABLE DRUGS FOR THE TREATMENT OF FOUR ORPHAN DISEASES: SICKLE CELL DISEASE,THALASSEMIA, HEMOCHROMATOSIS, AND ERYTHROPOIETIC PORPHYRIAS.FROM THE AVAILABLE WORLD MEDICAL LITERATURE ON THE FOUR DISEASES, DRUGS THAT ARE CLAIMED TO BE EFFECTIVE WILL BE EVALUATED AND CATEGORIZED AS TO THE EXTENT OF CLINICAL TESTING AND ANIMAL EXPERIMENTATION. THE THERAPEUTIC REVIEWS WILL BE EXAMINED BY A GROUP OF CONSULTANTS WHO WILL IN TURN SELECT APPROVABLE DRUGS FOR SUBMISSION TO THE ORPHAN PRODUCTS BOARD. PROMISING DRUGS THAT NEED ADDITIONALBUT MINIMAL RESEARCH WILL BE SELECTED FOR PHASE II STUDIES ON ANIMALS AND PATIENTS. IT IS EXPECTED THAT THIS PROJECT WILL HASTEN THE SELECTION PROCESS AND COMMERCIAL INTRODUCTION OF DRUGS FOR ORPHAN DISEASES IN GENERAL, PARTICULARLY OF HEREDITARY ERYTHROCYTIC DISEASES.