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Great News From HNF TRIAD Industry Partner — Applied Therapeutics

by | Aug 2, 2023 | 0 comments

Applied Therapeutics, Inc. has announced that their drug candidate, AT-007 (Govorestat), has received orphan medicinal product designation from the European Medicines Agency (EMA) for the treatment of Sorbitol Dehydrogenase (SORD) Deficiency. This designation reflects the high unmet medical need in SORD Deficiency and recognizes the benefit of Govorestat in reducing toxic sorbitol levels. New data published in the Journal of Clinical Investigation has further explained the molecular pathophysiology of SORD neuropathy and the role of sorbitol in neuronal toxicity. This is an important step for advancing regulatory initiatives in Europe and provides certain benefits and incentives for the development and marketing of the medicine.

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by | Sep 16, 2014 | , | 2 Comments

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by | Jul 7, 2014 | | 4 Comments

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by | May 19, 2014 | | 0 Comments

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Hot off the press

by | Feb 28, 2014 | | 0 Comments

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by | Feb 12, 2014 | | 6 Comments

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by | Sep 13, 2013 | , | 0 Comments

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by | Sep 6, 2013 | , | 0 Comments

HNF’s CSO (Chief Scientific Officer), Sean Ekins wrote a blog about his work with Allison Moore and her two “fighter Mom” friends Lori Sames and Jill Wood. He named his blog: “Rare disease heroes – Extraordinary collaborators we should be listening too.” Sean helped my friends and I write a paper called: “Multifaceted roles of ultra rare and rare disease patients/parents in drug discovery.” YES, Allison Moore is going to be published! It will be in Drug Discovery Today, soon. The link to the reprint is below.

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