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Drug Discovery

Drug Repurposing for Neurological Disorders

Neurological disorders are extremely widespread, affecting over 1 billion people worldwide, and have debilitating effects, impacting the brain, spinal cord, cranial nerves, and peripheral nerves. Despite this, many neurological disorders lack effective treatment. One strategy that offers an attractive way to find better therapies is drug repurposing. Also known as drug reprofiling or drug repositioning, drug repurposing identifies new purposes for pre-existing compounds, and offers reduced time frames, decreased costs, and improved success rates. While drug repurposing for neurological disorders has gained attention in the past decade, it is still an underused approach. In this article we will discuss drug repurposing, its benefits, its place in neurological drug research, and its recent success.

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HIV, COVID-19, Alzheimer’s: The Impact of The FDA’s Expedited Programs and What’s Next

The FDA’s emergency use authorization (EUA) of the COVID-19 vaccines has brought the approval process of vaccines and drugs into public consciousness, however, the FDA’s ability to streamline medical products is not new. In this article we will discuss the history of the drug development pipeline, and ways it can be improved.

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How Can a Drug Get to Market Faster? Explaining the FDA’s Expedited Drug Tracks

Getting a drug to market is notoriously difficult, taking 7-10 years and costing hundreds of millions of dollars. This, coupled with the Covid-19 pandemic’s impact on clinical trials, may make you wonder whether your drug is eligible for an expedited track. In this article we will discuss the current ways of expediting drugs, and which is best for your research.

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The Application of Hypersensitivity Assays to the Discovery of Therapeutics

The Application of Hypersensitivity Assays to the Discovery of Therapeutics

Using Humanization we can take advantage of the ancient biology between humans and other organisms to create stand-ins – patient avatars – for drug screening studies. In this blog, we will focus on models of inborn errors of metabolism (IEM), as these genetic conditions can lead to hypersensitivity to the metabolite. Since stressor condition hypersensitivity can be used to detect favorable drug effects, IEM model systems are ideal tools for phenotypic screens to find molecules that alleviate the metabolic stress occurring from the deficiency. We discuss the model organisms used in hypersensitivity screens, and why they are advantageous to drug discovery. Ultimately, showcasing this approach’s potential to be widely generalized to many genetic disorders.

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Let’s Fail Faster: Why Scientific Failures Should be Accepted as Progress

In the scientific community there is an emphasis on positive results: getting published, having a drug be approved, etc. However, crucial learning happens when experiments don’t work – in fact, these “failures” may be some of the most important learning experiences. In this article we discuss the need to shift the focus from failing less, to failing faster and some of the resources available to researchers so they can expedite their own work.

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The Path to Affordable Therapeutics in Rare Disease – Tackling Congenital Disorder of Glycosylation in the PMM2 gene.

Rare diseases are just that: rare. However, when combined rare diseases affect 3.5% – 5.9% of the worldwide population, and present a growing healthcare issue. In this article we talk with Dr Sangetha lyer about her work at the biotech Perlara and highlight their recent successes in a drug repurposing campaign for PMM2-CDG.

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