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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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Endometriosis partners to discover personalised treatments

Drug Discovery World

PrecisionLife and the University of Oxford have signed a data access agreement to license the Oxford Endometriosis Gene (OXEGENE) dataset to develop new personalised treatments for endometriosis patients. We hope that the analysis of our data will lead to the development of precision medicines to improve the lives of patients.”

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Can genetic data be a magic bullet for drug R&D?

pharmaphorum

The cost of testing per human genome in 2006 was approximately $14 million , and in less than two decades, an average consumer-purchased genetic test costs $100. The same is becoming true for the healthcare industry, and one of the first major breakthroughs in the area was the 100,000 Genomes Project.

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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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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 85
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One world, one health: The interconnected web of antimicrobial resistance

pharmaphorum

That’s according to a newly published report from the US National Academies of Sciences, Engineering, and Medicine, which calls for a One Health approach that recognises the scale of the challenge, and the interconnectivity of its potential solutions. Growing threat of AMR. million human infections and 35,900 deaths every year in the US.

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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