An Analysis of Metabolic Conditions and Current Therapeutic Approaches: A Systematic Review

An Analysis of Metabolic Conditions and Current Therapeutic Approaches: A Systematic Review | Research Square window.SnipcartSettings = { analytics: { enabled: false } }; (function() { var accessVector = localStorage.getItem('access_vector') || ''; window.dataLayer = window.dataLayer || []; if (accessVector) { window.dataLayer.push({ user: { profile: { profileInfo: { snid: accessVector } } } }); } })(); (function(w,d,s,l,i){w[l]=w[l]||[];w[l].push({'gtm.start':new Date().getTime(),event:'gtm.js'});var f=d.getElementsByTagName(s)[0],j=d.createElement(s),dl=l!='dataLayer'?'&l='+l:'';j.async=true;j.src='https://www.googletagmanager.com/gtm.js?id='+i+dl;f.parentNode.insertBefore(j,f);})(window,document,'script','dataLayer','GTM-K279D39R'); Browse Preprints In Review Journals COVID-19 Preprints AJE Video Bytes Research Tools Research Promotion AJE Professional Editing AJE Rubriq About Preprint Platform In Review Editorial Policies Our Team Help Center Sign In Submit a Preprint Cite Share Download PDF Systematic Review An Analysis of Metabolic Conditions and Current Therapeutic Approaches: A Systematic Review Hetvi Trivedi, Omar Fayaz, Christopher Korban, Christian Chung This is a preprint; it has not been peer reviewed by a journal. https://doi.org/ 10.21203/rs.3.rs-4228679/v1 This work is licensed under a CC BY 4.0 License Status: Posted Version 1 posted You are reading this latest preprint version Abstract This review rigorously investigates the evolving pharmacological landscape for metabolic disorders, focusing on Phenylketonuria (PKU), Inflammatory Bowel Disease (IBD), Galactosemia, Medium Chain Acyl CoA Dehydrogenase Deficiency (MCADD), and Prader-Willi Syndrome (PWS). Adhering to PRISMA guidelines, we meticulously searched databases such as NHS, Rare Diseases, Pubchem, Clinical Trials, DrugBank, PubMed, and Mayo Clinic, spanning from January 2000 to December 2023. Our objective was to evaluate the efficacy and potential of both FDA-approved drugs and those in clinical trials. The review revealed significant advancements in the treatment of disorders like PKU, with drugs like Kuvan facilitating improved metabolic functioning. In IBD, treatments have progressed to specifically target inflammatory pathways using drugs like Azathioprine and Mesalazine, shifting away from broader immunosuppressants. Galactosemia and MCADD, disorders without definitive cures, have shown potential in clinical trials with investigational drugs like AT-007 and Triheptanoin. Particularly intriguing are the ongoing trials for PWS, a complex syndrome, where Oral NNZ-2591 and Oxytocin demonstrate promising potential. These findings highlight the critical importance of specialized treatment strategies, tailored to address the unique metabolic challenges posed by each disorder. The diverse nature of these disorders necessitates a multifaceted approach, combining current scientific knowledge with innovative drug development. This study not only contributes to a better understanding of existing treatments but also emphasizes the need for continued research and development to uncover more effective and personalized therapeutic options for managing these varied metabolic disorders. Clinical Pharmacology Biotechnology and Bioengineering Endocrinology & Metabolism Full Text Additional Declarations The authors declare potential competing interests as follows: For H.T., O.F., C.K., C.C., and authors that are affiliated with Nexabio Venture Solutions LLC: Nexabio Venture Solutions LLC is a corporation focused on AI-driven drug discovery. We specialize in repurposing abandoned therapeutics with an emphasis on developing computational and predictive modeling for enhanced understanding of diseases and drugs. Nexabio Venture Solutions LLC holds proprietary algorithms in the field of drug repurposing for several disease states and drug targets. The authors are engaged in creating and applying AI models to facilitate drug discovery and to provide greater insights into a broad array of conditions, including metabolic diseases. O.F., C.K., C.C., and H.T. are affiliated with Nexabio Venture Solutions LLC, and have contributed to the research and development of the disease/drug models discussed in this review. No other conflicts are reported. Cite Share Download PDF Status: Posted Version 1 posted You are reading this latest preprint version Research Square lets you share your work early, gain feedback from the community, and start making changes to your manuscript prior to peer review in a journal. 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