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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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How Pharmacogenomics may finally realise its promise

pharmaphorum

Pharmacogenomics, the understanding of how genes impact an individual’s response to medications, provides a potential outlet to better optimised therapies. There have been great strides made in genomics to refine tools that determine the safest, most effective course of treatments for patients across a number of disease states.

Genome 96
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Rare Disease Diagnosis: Why Tackling the Genomic Analysis Bottleneck is Key to Advancing Precision Medicine

XTalks

For more information on tackling this “genomic analysis bottleneck,” watch this on-demand webinar. How many genes need to be scanned in order to determine the presence of a genetic disease? It also slows the progress of identifying new disease gene associations in very rare conditions.

Genome 98
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HFpEF vs. HFrEF: How To Improve Heart Failure Drug Development

XTalks

New advances in heart failure genomics are helping to address this challenge. Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. Watch this on-demand webinar to hear from these experts.

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Why a recent advancement is a giant leap for human genomics

Drug Discovery World

The first complete, gapless sequence of a human genome was published 1 April 2022 in a special issue of the journal Science 1. While The Human Genome Project mapped about 92% of the human genome two decades ago, sequencing the last 8% of the genome proved highly challenging.

Genome 52
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How biomarkers can help discover new treatments for women’s health

Drug Discovery World

RA: You’ve recently announced a collaboration with the University of Oxford to access genotype data on women with endometriosis. SG: Endometriosis is a common disease that causes chronic pain and reduced fertility, reducing the quality of life of over 200 million women around the world.

Genome 72