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AI Uses Zero-Shot Learning to Find Existing Drugs for Treating Rare Diseases

Nvidia Developer: Drug Discovery

A groundbreaking drug-repurposing AI model could bring new hope to doctors and patients trying to treat diseases with limited or no existing treatment options. A groundbreaking drug-repurposing AI model could bring new hope to doctors and patients trying to treat diseases with limited or no existing treatment options.

Disease 144
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As ALS research booms, one treatment center finds itself in the spotlight

BioPharma Drive: Drug Pricing

The Healey center is at the front of ALS research and care, earning acclaim from patients, doctors and scientists. Still, the complexities of the disease and of drug development have brought hard-felt losses.

Research 142
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Researchers design genetic therapy to prevent brain deterioration

Drug Discovery World

A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. The researchers have shown that problems with calcium underlie these progressive diseases.

Therapies 147
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Researchers identify potential new genetic target for cancer drugs

Drug Discovery World

UK researchers have mapped the exact variants in a gene that dramatically increase a person’s risk of developing cancer, paving the way for targeted treatment across diverse populations. The research team tested all 18,108 possible DNA changes in the BAP1 gene using ‘saturation genome editing’.

Research 130
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With retinal images and genetic data, researchers predict cardiovascular, metabolic, and other disease risks

Broad Institute

They found significant associations between the thinning of different retinal layers and increased risk of developing ocular, cardiac, pulmonary, metabolic, and neuropsychiatric diseases and identified genes that are associated with retinal layer thickness. Their findings are published in Science Translational Medicine. “We

Disease 124
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Novel biosensor could help optimise cancer treatment

Drug Discovery World

This biosensor has the potential to aid the development of new treatments for DNA damage-related diseases by revealing how our bodies repair damaged DNA. The post Novel biosensor could help optimise cancer treatment appeared first on Drug Discovery World (DDW).

Treatment 130
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A smartphone app for the self-management of Perthes’ Disease – a blog for Rare Disease Day 2024

On Medicine

What is Perthes’ disease? Perthes’ Disease is a rare condition that affects the developing femoral head (the “ball” of the “ball and socket” hip joint) of a child due to poor blood supply. This in turn can cause significant pain for children with Perthes’ disease and limit their activity levels significantly.

Disease 109