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Applied Therapeutics to Present Cross-Sectional Analysis of Galactosemia Disease Progression at 2021 Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics


Published: Apr 07, 2021
NEW YORK, April 07, 2021 (GLOBE NEWSWIRE)
Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced a poster presentation at the upcoming 2021 Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics (ACMG) taking place April 13-16, 2021. The abstract and presentation include a cross-sectional analysis of nineteen pediatric patients with Classic Galactosemia, characterizing progressive worsening of the central nervous system (CNS) phenotype with age.
“We are pleased to share this important data with the medical community,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “The severity and significant decline in CNS outcomes with age underscores the urgency for a disease-modifying therapy to halt or pre ....

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Applied Therapeutics to Host Virtual Rare Disease Forum - Mar 16, 2021


Published: Mar 16, 2021
NEW YORK, March 16, 2021 (GLOBE NEWSWIRE)
Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced it will host a Virtual Rare Disease Forum on Tuesday, March 23
rd, 2021 from 1:00pm to 3:00pm ET. The event will highlight Applied Therapeutics’ development programs in Galactosemia, SORD Deficiency, and PMM2-CDG. The agenda for the event will include:
Update on ACTION-Galactosemia Clinical Development Program
Overview of SORD Deficiency, presented by Stephan L. Züchner, MD, PhD, Professor and Department Chair of Human Genetics at the University of Miami Miller School of Medicine ....

United States , Joseph Muenzer , Maeve Conneighton , Shoshana Shendelman , University Of North Carolina Children Research Institute , Clinical Development Program , Virtual Rare Disease Forum On , University Of Miami Miller School Medicine , Deficiency Clinical Development , Therapeutics Inc , Virtual Rare Disease Forum , Applied Therapeutic , Galactosemia Clinical Development , Department Chair , Human Genetics , Miami Miller School , Clinical Development , Pediatric Genetics , North Carolina Children , Development Program , Diabetic Cardiomyopathy , Applied Therapeutics To Host Virtual Rare Disease Forum Mar 16 , ஒன்றுபட்டது மாநிலங்களில் , பல்கலைக்கழகம் ஆஃப் வடக்கு கரோலினா குழந்தைகள் ஆராய்ச்சி நிறுவனம் , மருத்துவ வளர்ச்சி ப்ரோக்ர்யாம் , மெய்நிகர் ரேர் நோய் மன்றம் ஆன் ,