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Leveraging Genetic Testing for Enrolling Rare Disease Trials

Worldwide Clinical Trials

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

Genetics 165
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$8 million awarded to landmark genetic research project

Drug Discovery World

Landmark genetic research could allow doctors to accurately predict whether a patient is at risk of developing common diseases, decades before any symptoms would become evident. He’s going to accelerate personalised medicine based on an individual’s genome.

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Researchers identify large genetic changes that contribute to dementia risk

Medical Xpress

Scientists at the National Institutes of Health have identified new genetic risk factors for two types of non-Alzheimer's dementia. These findings were published in Cell Genomics and detail how researchers identified large-scale DNA changes, known as structural variants, by analyzing thousands of DNA samples.

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UK agency pilots biobank to study links between genetics and drug side effects

Pharmaceutical Technology

The Medicines and Healthcare products Regulatory Agency (MHRA) aims to launch a pilot genetic biobank that will gather patient data to associate drug-related adverse events to their genetic makeup. The Yellow Card biobank will launch as a joint venture with the UK-government funded entity Genomics England on June 1.

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Emmes and MedGenome Launch Genomics Strategic Partnership Focused on Advancing Rare Disease Research

Pharma Mirror

Rockville, Emmes, a global, full-service Clinical Research Organization dedicated to supporting the advancement of public health and biopharmaceutical innovation, today announced a partnership with MedGenome aimed at accelerating breakthrough treatments, powered by human genomics, for rare disease patients.

Genome 130
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Hopewell Therapeutics raises funds for genomic medicines development

Pharmaceutical Technology

Biotechnology company Hopewell Therapeutics has raised $25m in seed financing to accelerate the development of next-generation lipid nanoparticles for targeted delivery of genomic medicines. Hopewell Therapeutics is engaged in discovering, synthesising and developing advanced ttLNPs to provide next-generation genomic medicines.

Genome 130
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National Academies calls for transforming use of racial and ethnic labels in genetics research

STAT News

Saying genetics researchers inconsistently and inappropriately use racial and ethnic labels that fail to capture the complex patterns of human genetic variation, the National Academies of Sciences, Engineering, and Medicine issued a report Tuesday calling for a transformation in how such descriptors are used.