Drug Repurposing News and Research

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FDA-approved osteoporosis drug may treat rare genetic heart disease

A research team co-led by scientists at the University of Arizona College of Medicine – Tucson found that an osteoporosis drug might counter a rare genetic mutation underlying a type of heart disease.

18 Feb 2025

New study identifies potential drug targets for heart failure

Heart failure remains a substantial burden for patients due to its high prevalence and limited therapeutic options. Heart failure is classified into two major clinical subtypes- heart failure with preserved ejection fraction (HFpEF) and heart failure with reduced ejection fraction (HFrEF).

6 Feb 2025

AI predicts effective treatment for rare disease using existing medications

After combing through 4,000 existing medications, an artificial intelligence tool helped uncover one that saved the life of a patient with idiopathic multicentric Castleman's disease (iMCD).

5 Feb 2025

AI technology uncovers genetic factors and treatment options for Parkinson's

Researchers from the Cleveland Clinic Genome Center have successfully applied advanced artificial intelligence (AI) genetics models to Parkinson's disease. Researchers identified genetic factors in progression and FDA-approved drugs that can potentially be repurposed for PD treatment.

28 Jan 2025

Study reveals potential drug links to Dementia risk

Findings from a large-scale review indicate certain medications may influence dementia risk, offering insights for prevention and drug repurposing strategies.

23 Jan 2025

New computational pipeline identifies key biomarkers for Alzheimer's disease

Researchers at Columbia University Mailman School of Public Health have developed a novel computational pipeline designed to identify protein biomarkers associated with complex diseases, including Alzheimer's disease (AD).

6 Dec 2024

Scientists link proteins to Alzheimer’s through cutting-edge genetic method

Researchers have developed MR-SPI, a novel method integrated with AlphaFold3, to identify causal protein biomarkers and predict their 3D structural changes in Alzheimer’s disease. This breakthrough offers insights into disease mechanisms and potential therapeutic targets.

5 Dec 2024

Scientists uncover cerebrospinal fluid markers for Alzheimer's detection and treatment

Researchers map the unique genetic regulation of proteins in human cerebrospinal fluid, identifying potential targets for Alzheimer’s treatments.

12 Nov 2024

New pipeline analyzes drug responses in primary tumors

In a major leap toward more effective cancer treatments, researchers at the University of Oulu have developed an innovative pipeline designed to directly analyze drug responses in primary patient tumor samples.

1 Nov 2024

Drug repurposing offers hope for SYNGAP1 patients

The SynGAP Research Fund 501(c)(3) has awarded a pair of grants to Dr. Clement Chow, a geneticist and Associate Professor in the Department of Human Genetics at the University of Utah. These grants are crucial steps in advancing therapeutic development for SYNGAP1-Related Disorders (SRD).

30 Oct 2024

Groundbreaking discovery offers hope for patients suffering from toxic epidermal necrolysis

A global team of researchers at the Max Planck Institute of Biochemistry has made a groundbreaking discovery that saves the lives of patients suffering from toxic epidermal necrolysis.

16 Oct 2024

Harnessing the power of miRNA for cancer diagnosis and treatment

This study is led by Dr. Shiwei Duan (Department of Clinical Medicine, School of Medicine, Hangzhou City University, Hangzhou, Zhejiang, China). His team analyzed literature on miRNA, summarizing the close relationship between these small molecules and cancer. miRNA is a type of non-coding RNA that does not encode proteins, but can target more than 60% of human protein-coding genes.

15 Oct 2024

Differential gene expression linked to alcohol use disorder, offering new treatment possibilities

Researchers identified key gene expression differences in addiction-relevant brain regions that provide new insights into alcohol use disorder and potential therapeutic targets.

14 Oct 2024

New deep-learning framework identifies non-addictive pain relief options

An estimated one in five Americans live with chronic pain and current treatment options leave much to be desired.

7 Oct 2024

A randomized trial of mexiletine and lamotrigine in adults with non-dystrophic myotonia

The study, published in Lancet Neurology, detailed the "head-to-head" trial implemented by the researchers to test two drugs, mexiletine and lamotrigine, on people with the condition.

27 Sep 2024

TxGNN improves drug repurposing by predicting treatments for rare diseases with no approved therapies

A new model, TxGNN, revolutionizes zero-shot drug repurposing by predicting treatments for diseases with no known therapies, offering improved accuracy and trust via interpretable AI explanations.

27 Sep 2024

New AI tool revolutionizes drug discovery for rare diseases

There are more than 7,000 rare and undiagnosed diseases globally.

25 Sep 2024

Combination therapy incorporating three existing drugs shows potential for treating retinal degenerations

Drug repurposing shows promise in the treatment of retinal degenerations, according to a new study by an international team of researchers, published in Nature Communications.

22 Jul 2024

Existing drug shows promise for treating common liver disease

The University of Barcelona has led a study that suggests using the drug known as pemafibrate to treat liver disease associated with metabolic disorders, the most common liver pathology in the world, which affects one in four people. The drug has long been marketed in Japan for another use: improving blood lipid levels in patients with hyperlipidaemia, a common condition in diabetics. Now, however, it could help address this serious liver disease, which still has no specific treatment.

11 Jul 2024

Repurposing pemafibrate to treat liver disease associated with metabolic disorders

10 Jul 2024