Galactosemia

July 10, 2024
Safety, Pharmacokinetics, and Pharmacodynamics of the New Aldose Reductase Inhibitor Govorestat (AT-007) After a Single and Multiple Doses in Participants in a Phase 1/2 Study
Riccardo Perfetti, MD, PhD, Evan Bailey, MD, Stella Wang, MPH, MS, Richard Mills, PhD, Ramon Mohanlal, MD, PhD, MBA, Shoshana Shendelman, PhD
Journal of Clinical Pharmacology

August 26, 2024
Towards an Advanced Understanding of the Pathogenesis of CNS Complications Associated with Galactosemia: Targeting Galactitol for Pharmacological Intervention with Govorestat, a Novel Aldose-Reductase Inhibitor
Riccardo Perfetti, Susan E Waisbren, Jonathan W Mink, Evan Bailey, Shoshana Shendelman
Journal of Pharmacological Reports

September 4, 2024
Development of Govorestat (AT-007), the First Potential Treatment for Patients with Classic Galactosemia
Presented by Barbara Burton, MD, Professor of Pediatrics, Genetics, Genomics and Metabolism at the Northwestern University Feinberg School of Medicine, Riccardo Perfetti, MD, PhD, Chief Medical Officer, Applied Therapeutics, and Evan Bailey, MD, Vice President Clinical Development, Applied Therapeutics
2024 Annual Symposium of the Society for the Study of Inborn Errors of Metabolism

November 6, 2024
Results of the ACTION-Galactosemia Kids Study to Evaluate the Effects of Govorestat in Pediatric Patients with Classic Galactosemia
Evan Bailey, MD, Han Phan, MD, Ayesha Ahmad, MD, Janet Thomas, MD, Elizabeth G. Ames, MD, Amanda B. Pritchard, MD, Shane C. Quinonez, MD, Stella Wang, MS, MPH, Caleb Dayley, MS, Andrew Salt, MS, Christina Pick, MS, Abe Durrant, MS, Samuel Johnson, MS, Jessie Nicodemus-Johnson, PhD, Samuel P. Dickson, PhD, Riccardo Perfetti, MD, PhD, Suzanne B. Hendrix, PhD, and Shoshana Shendelman, PhD
Journal of Clinical Pharmacology

Sorbitol Dehydrogenase (SORD) Deficiency

May 4, 2020
Biallelic mutations in SORD cause a common and potentially treatable hereditary neuropathy with implications for diabetes
Andrea Cortese, Yi Zhu, Adriana P. Rebelo, Sara Negri, Steve Courel, Lisa Abreu, Chelsea J. Bacon, Yunhong Bai, Dana M. Bis-Brewer, Enrico Bugiardini, Elena Buglo, Matt C. Danzi, Shawna M. E. Feely, Alkyoni Athanasiou-Fragkouli, Nourelhoda A. Haridy, Inherited Neuropathy Consortium, Rosario Isasi, Alaa Khan, Matilde Laurà, Stefania Magri, Menelaos Pipis, Chiara Pisciotta, Eric Powell, Alexander M. Rossor, Paola Saveri, Janet E. Sowden, Stefano Tozza, Jana Vandrovcova, Julia Dallman, Elena Grignani, Enrico Marchioni, Steven S. Scherer, Beisha Tang, Zhiqiang Lin, Abdullah Al-Ajmi, Rebecca Schüle, Matthis Synofzik, Thierry Maisonobe, Tanya Stojkovic, Michaela Auer-Grumbach, Mohamed A. Abdelhamed, Sherifa A. Hamed, Ruxu Zhang, Fiore Manganelli, Lucio Santoro, Franco Taroni, Davide Pareyson, Henry Houlden, David N. Herrmann, Mary M. Reilly, Michael E. Shy, R. Grace Zhai, Stephan Zuchner
Nature Genetics

Diabetic Cardiomyopathy (DbCM) 

April 8, 2024
A Selective Aldose Reductase Inhibitor (AT-001) For the Treatment of Diabetic Cardiomyopathy: Primary Results of the Phase 3 Randomized Controlled ARISE-HF Study
Presented by Riccardo Perfetti, MD, PhD, Chief Medical Officer, Applied Therapeutics
2024
American College of Cardiology Annual Scientific Session

July 9, 2024
Randomized Trial of a Selective Aldose Reductase Inhibitor in Patients with Diabetic Cardiomyopathy
James L. Januzzi, MD, Javed Butler, MD, MPH, MBA, Stefano Del Prato, MD, Justin A. Ezekowitz, MB, BCH, Nasrien E. Ibrahim, MD, MPH, Carolyn S.P. Lam, MBBS, PHD, Gregory D. Lewis, MD, Thomas H. Marwick, MBBS, PHD, MPH, Riccardo Perfetti, MD, PHD, Julio Rosenstock, MD, Scott D. Solomon, MD, W.H. Wilson Tang, MD, Faiez Zannad, MD, PHD
Journal of the American College of Cardiology

November 2, 2022
Aldose Reductase Inhibition by AT-001 Alleviates Fibrosis and Adverse Remodelling in Diabetic Cardiomyopathy by Reducing Myocardial Fatty Acid Oxidation
Presented by Keshav Gopal, Qutuba G Karwi, S. Amirhossein Tabatabaei-Dakhili, Cory S Wagg, Liyan Zhang, Qiuyu Sun, Christina T Saed, Sai Panidarapu, Riccardo Perfetti, Ravichandran Ramasamy, John R Ussher, Gary D Lopaschuk
American Heart Association (AHA) Scientific Session 2022

Diabetic Cardiomyopathy Is Associated with Worsening Quality of Life Long Before the Development of Overt Heart Failure: Lessons from the Baseline Analysis of the ARISE-HF Trial
Presented by Alessia Urbinati, Riccardo Perfetti, Stella Wang, Sariah Persaud, Shoshana Shendelman, James L Januzzi Jr.
American Heart Association (AHA) Scientific Session 2022