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A transatlantic collaboration is uncovering the role of key genetic variants in common diseases like type 2 diabetes and obesity

Broad Institute

By Lucy Mutz November 4, 2024 Credit: Juliana Sohn Kasper Lage is managing director of the Novo Nordisk Foundation Center for Genomic Mechanisms of Disease. The overall goal is to translate genetic findings into biological mechanisms underlying disease that could be targeted with new medicines.

Disease 72
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Rilzabrutinib Cuts Flares in IgG4-Related Disease; Fast Track Granted in US

The Pharma Data

The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.

Disease 40
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Building better brain models for Parkinson’s disease and beyond

Drug Target Review

The search for effective treatments for neurodegenerative diseases like Parkinson’s disease has long been hindered by the brain’s complexity and the absence of adequate models for drug discovery. “For diseases like Parkinson’s, it’s more than sufficient,” he explains.

Disease 52
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The future of CNS drug development: signs of real progress

Drug Target Review

Disorders such as Alzheimer’s disease , primary glioblastoma and amyotrophic lateral sclerosis (ALS) all affect the CNS and are considered fatal, while more common conditions, including depression , strokes and epilepsy , require long-term treatments. Hundreds of life-altering conditions attack the central nervous system (CNS).

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Prime editing treats childhood brain disease in mice

Broad Institute

There is no cure or effective treatment for this rare genetic disease, but new research suggests a potential path to one.& & The new study, published in Cell , is the first time prime editing has been used to treat a neurological disease in animals, offering hope for treating people with AHC and other genetic brain disorders.

Disease 141
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A spatial approach to understanding drug dynamics using mass spectrometry imaging

Drug Target Review

Bioanalysis 2019, 11 (11), 1099–1116. Multimodal Imaging Distribution Assessment of a Liposomal Antibiotic in an Infectious Disease Model. Quantification and Assessment of Detection Capability in Imaging Mass Spectrometry Using a Revised Mimetic Tissue Model. Cheng S-H, Groseclose MR, Mininger C, et al. Release 2022, 352 , 199–210.

Drugs 52
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Nobel Prize celebrates AI’s role in protein structure innovation

Drug Target Review

Its applications include studying disease pathology to develop targeted therapeutics, visualising enzymes that can decompose plastics, and engineering solutions to antibiotic resistance, among many others. David Baker is listed as an inventor on many patents, focused on compositions and methods for treating particular diseases.