Introduction: Cerebrospinal fluid (CSF) is used for measuring reliable Brain serotonin levels. However, it involves an invasive procedure especially for Monoamine Oxidase Deficient (MAOD) children. Audhya et al. (2012) reported that blood platelet serotonin level is well correlated with CSF serotonin level more than blood plasma serotonin. For measuring serotonin levels (a crucial biomarker) in […]
Patient-derived induced pluripotent stem cells (iPSCs) are an important tool for studying rare diseases and developing new therapies. Previously, MAODF had an iPSC line generated from a MAO-A/B patient and an iPSC line from a healthy parent as control. These cell lines have been successfully differentiated into nerve cells and are being used in ongoing […]
MAODF continues to make important progress in identifying potential treatments for MAO Deficiency. Following an AI-driven screening of approximately 40,000 compounds conducted by Unravel Biosciences, 14 promising drug candidates were selected for further testing. These compounds are currently being evaluated in Unravel’s Xenopus (frog) model of MAO Deficiency. At the same time, MAODF is collaborating […]
(AI-Unbiased drug screening & known neurotransmitter modulators) MAODF is pursuing multiple approaches to identify potential therapies for MAOD, including AI-driven unbiased drug screening and evaluation of known neurotransmitter-regulating compounds. In collaboration with Unravel Biosciences, approximately 40,000 compounds were screened using their proprietary BioNAV platform, which analyzes RNA sequence (RNAseq) profiles to identify drugs capable of […]
Over the past three years, MAODF has been working with research teams at University of Texas Southwestern Medical Center and The Ohio State University to develop a gene therapy approach for patients with Monoamine Oxidase Deficiency (MAOD). Gene therapy is considered a promising long- term strategy because it may one day provide a more permanent […]
FDA grants Monoamine Oxidase Deficiency Orphan Drug Designation Galveston, Texas — Families affected by monoamine oxidase deficiency (MAOD), a rare neurodevelopmental disorder, have historically faced delayed diagnosis, limited clinical guidance and scant treatment options. MAOD is an X-linked genetic condition caused by impaired activity or the total lack of the monoamine oxidase A and/or monoamine […]