Developing transformative treatments for patients with high unmet medical need

Patient-First Approach

If you or a loved one receives a diagnosis for which there are no treatment options, it can be devastating. At Applied Therapeutics, we are dedicated to creating transformative treatments for patients who have no approved therapies for their conditions. By applying the right science and the right development strategies, Applied Therapeutics aims to rapidly advance new treatments for patients with high unmet need conditions.

Target Disease Areas






SORD Deficiency





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Clinical Trials

Applied Therapeutics is committed to developing transformative treatments to treat debilitating diseases with high unmet medical need.

Clinical trials are conducted at approved study sites such as hospitals, universities, doctors’ offices, and community clinics, in which people volunteer to help researchers learn about new and existing treatments. Clinical trials are traditionally divided into the following steps, called “phases”:

  • Phase 1: measures safety and how the body breaks down the study treatment
  • Phase 2: measures safety and effectiveness or the ability of a trial treatment to produce a beneficial change
  • Phase 3: further measures safety and effectiveness of the trial treatment

Our lead investigational treatments, govorestat and AT-001, are being studied in clinical trials for the potential treatment of Galactosemia and Diabetic Cardiomyopathy. These trials help us test the treatment’s safety and efficacy. If the data shows the study treatment is safe and effective, it may be approved for use by prescription.

Patient Advocacy

At Applied Therapeutics, the patient advocacy team is passionate about educating and supporting patients, families and caregivers. Our organization applauds the patient advocacy organizations dedicated to supporting people living with serious illnesses.

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1.         Ultra Rare Advocacy, About Clinical Trials: