Acknowledgement: This article was originally written by Gillian Sapia, a mother of a child living with galactosemia, for Underground Rare Road. To see the original article, please click here. When…
Applied Therapeutics has encountered a significant setback in its quest to bring a new treatment to patients with a rare metabolic disorder. According to Biospace.com, the company announced that its…
In a news release on the company’s website, biopharmaceutical company Applied Therapeutics, Inc. shared that positive data was available from the Phase 3 ACTION-Galactosemia Kids trial. Within the trial, researchers…
Jim Kelly and his wife Maria still remember the day they received a call from the hospital telling them that according to test results, their daughter Adriana, now age…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
According to a relatively recent news release from biopharmaceutical company Applied Therapeutics, Inc., AT-007 received Fast Track designation from the FDA. This therapy, which has also received both Orphan Drug…
Galactosemia Galactosemia is a rare disease which affects the metabolic system. It causes the body to not be able to process galactose properly. Galactose is a simple sugar present in…
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