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Find Novel Drugs for Rare Diseases and Disorders under Development in UNITED STATES
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Olipudase Alfa is a Enzyme drug candidate, which is currently being evaluated in clinical studies for the treatment of Niemann-Pick Disease, Type A.
Details :
RB001 is a Cell and Gene therapy drug candidate, which is currently being evaluated in clinical studies for the treatment of Phelan-McDermid Syndrome.
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Probucol is a Other Small Molecule drug candidate, which is currently being evaluated in clinical studies for the treatment of Mitochondrial Diseases.
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Dichloroacetate is a Other Small Molecule drug candidate, which is currently being evaluated in clinical studies for the treatment of Pyruvate Dehydrogenase Complex Deficiency Disease.
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Cysteine is a Other Small Molecule drug candidate, which is currently being evaluated in clinical studies for the treatment of Homocystinuria.
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Doxecitine is a Other Small Molecule drug candidate, which is currently being evaluated in clinical studies for the treatment of Thymidine Kinase 2 Deficiency.
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KL003 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in clinical studies for the treatment of beta-Thalassemia.
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Pozelimab is a Antibody drug candidate, which is currently being evaluated in clinical studies for the treatment of Hemoglobinuria, Paroxysmal.
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