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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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LabCorp Backs Resolution Bioscience’s Liquid Biopsy Test for NSCLC

XTalks

The leading global life science company is set to introduce Resolution Bio’s ctDx Lung assay , a non-invasive liquid biopsy test for patients with non-small cell lung cancer (NSCLC). The panel of genes in the test only include known NSCLC-associated genes, making the test specific to the disease. Personalized Approach.

Gene 105
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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. The program is aiming to genotype roughly 150,000 participants over the next few years to demonstrate the impact personalised testing can have on a large scale.

Genome 95
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BridgeBio Pharma’s Nulibry Approved as First Treatment for Molybdenum Cofactor Deficiency Type A

XTalks

MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene. In the analysis involving 31 patients, 13 were treated with Nulibry and compared to 18 genotype-matched untreated patients from the history control group. What is Molybdenum Cofactor Deficiency Type A?

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Global Roundup: Overland Pharma Launches in China to Provide Access to Breakthrough Therapies

The Pharma Data

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

In-Vivo 52
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HFpEF vs. HFrEF: How To Improve Heart Failure Drug Development

XTalks

From a total of 30 single-nucleotide polymorphisms (SNPs) in genes previously identified as being associated with blood pressure, many had a relatively neutral effect on the risk of coronary artery disease. Figure 2: Using drug-target MR to validate sympathetic nervous system genes involved in heart failure.

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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