Remove Containment Remove Development Remove Genetics Remove Genotype
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Why early participant engagement is now a top priority in genetic disease research

pharmaphorum

In 2016, scientists behind a study called the Resilience Project analysed genetic data from 589,000+ people and found 13 adults who carried genetic variants that should have resulted in serious – even deadly – childhood disease, but who were apparently healthy. First, start co-development with participants early on in the process.

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

XTalks

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. The reverse could also be true whereby the development of coronary artery disease may impact heart rate. HFrEF vs. HFpEF.

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FDA Makes Low-Risk Determination for Marketing of Products from Genome-Edited Beef Cattle After Safety Review

The Pharma Data

The IGA results in the equivalent genotype (genetic make-up) and short-hair coat trait seen in some conventionally bred cattle, known as a “slick” coat. This is the FDA’s first low-risk determination for enforcement discretion for an IGA in an animal for food use. Solomon, D.V.M.,

Genome 52
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BridgeBio Pharma’s Nulibry Approved as First Treatment for Molybdenum Cofactor Deficiency Type A

XTalks

The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. The approval was granted to BridgeBio Pharma, Inc.

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Adial Pharmaceuticals Provides Update on its ONWARD(TM) Pivotal Phase 3 Trial of AD04 for Treatment of Alcohol Use Disorder

The Pharma Data

(NASDAQ:ADIL; ADILW) , a clinical-stage biopharmaceutical company focused on the development of treatments for addictions, today provided an update on its landmark ONWARD Phase 3 pivotal trial. Adial Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the development of treatments for addictions.

Trials 52
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Rare Disease Diagnosis: Why Tackling the Genomic Analysis Bottleneck is Key to Advancing Precision Medicine

XTalks

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases.

Genome 98