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Find Novel Drugs for Rare Diseases and Disorders under Development
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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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Under the partnership, Burjeel and BridgeBio will revolutionize the field of early diagnosis and treatment of rare diseases or disorders in the UAE and the region.
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DFMO (eflornithine) is a novel cytostatic agent that irreversibly inhibits ornithine decarboxylase, a key enzyme in mammalian polyamine biosynthesis that is up-regulated in certain types of cancer.
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Sanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.
