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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 148
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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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Combination therapy halts lung cancer tumour growth in mice

Drug Discovery World

A pathway to better therapies. The findings suggest a pathway to better therapies for one of the least treatable forms of cancer. High levels of SMYD3 are associated with more invasive disease, increased resistance to alkylating chemotherapy and worse prognosis. This has not changed for 30 years.

Therapies 130
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Zebrafish for Disease Modeling and Drug Screening in Pediatric Cancers

biobide

Pediatric cancers exhibit differences at the genetic level compared to the same form of adult disease, which may influence the selected treatment. Children and adolescents lack robust preclinical models to replicate the pathologies and provide precise and targeted therapies.

Disease 59
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How AI is accelerating the future of precision medicine

Drug Target Review

How does the integration of Real-World Data (RWD) with genomic biomarker data contribute to a more comprehensive understanding of disease progression and treatment response? This one size fits all approach to drug prescribing still pervades in mostly all therapy areas except oncology. Most drugs do not work in all people.

Doctors 109
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Sloane’s 1st ASO treatment

KIF1A

Fortunately, Sloane does not currently have epilepsy, and Megan hopes this treatment will prevent it’s onset as her disease progresses. On the day of the treatment, they checked into the hospital where Sloane was administered sedation medication. During sedation, Sloane also had an MRI to assess for cerebral atrophy.

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New Hope Against Diseases Marked by Progressive Scarring of Lung Tissue

The Pharma Data

13 in the New England Journal of Medicine , involved patients with high blood pressure in the lungs caused by interstitial lung disease (ILD). It can have a range of causes, from smoking, to occupational exposure to toxins like asbestos, as well as autoimmune diseases like rheumatoid arthritis. The study, published online Jan.

Disease 52