Vafidemstat: a lysine-specific demethylase 1A inhibitor that shows promise for treating neuropsychiatric disorders

The enzyme KDM1A encoded by <i>KDM1A</i> and also known as lysine-specific demethylase 1 (LSD1) plays important roles in the development and function of the brain. Vafidemstat is a KDM1A inhibitor that has shown beneficial effects in preclinical and clinical trials for the treatment of neuropsychiatric disorders, including borderline personality disorder (BPD), autism spectrum disorder (ASD), and attention-deficit hyperactivity disorder (ADHD). This article reviews available preclinical and clinical data on the use of vafidemstat in the treatment of these disorders. Vafidemstat appears to be a potential new and safe drug for the treatment of these disorders.

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Journal
Epigenetics Reports
Published
2025-09-04
DOI
https://doi.org/10.1080/28361512.2025.2552947
Primary Topic
Epigenetics and DNA Methylation
Type
article
Field-Weighted Citation Impact
0.00

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article

Vafidemstat: a lysine-specific demethylase 1A inhibitor that shows promise for treating neuropsychiatric disorders

Dennis R. Grayson, Jacob Peedicayil
Epigenetics Reports
Epigenetics and DNA Methylation
article

Vafidemstat: a lysine-specific demethylase 1A inhibitor that shows promise for treating neuropsychiatric disorders

Dennis R. Grayson, Jacob Peedicayil
article en

Abstract

The enzyme KDM1A encoded by <i>KDM1A</i> and also known as lysine-specific demethylase 1 (LSD1) plays important roles in the development and function of the brain. Vafidemstat is a KDM1A inhibitor that has shown beneficial effects in preclinical and clinical trials for the treatment of neuropsychiatric disorders, including borderline personality disorder (BPD), autism spectrum disorder (ASD), and attention-deficit hyperactivity disorder (ADHD). This article reviews available preclinical and clinical data on the use of vafidemstat in the treatment of these disorders. Vafidemstat appears to be a potential new and safe drug for the treatment of these disorders.

Epigenetics ReportsVol. 3(1)
Illinois College (US), Christian Medical College, Vellore (IN), University of Illinois Chicago (US), Christian Medical College (IN), University Psychiatric Hospital (SI)
National Institute on Alcohol Abuse and Alcoholism
Openalex Percentile: Top 79%
Epigenetics and DNA Methylation
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Vafidemstat: a lysine-specific demethylase 1A inhibitor that shows promise for treating neuropsychiatric disorders — Dennis R. Grayson, Jacob Peedicayil · Epigenetics Reports (2025) | TGRS Research Map | TGRS