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Why early participant engagement is now a top priority in genetic disease research

pharmaphorum

The DNA of these so-called ‘genetic superheroes’ may contain clues about how to treat severe disease. Whether these people were truly naturally resistant to severe disease, and why that might be the case, remains a mystery. A decidedly contentious area in genomics is the patient’s ‘right not to know’.

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

XTalks

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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

XTalks

million people in the US, yet there is no treatment for the most common form of the disease: heart failure with preserved ejection fraction (HFpEF). There are different subtypes of heart failure that have different clinical presentations.”. New advances in heart failure genomics are helping to address this challenge.