How much enterprise has the USA lost in just one year?
A treatment to end suffering and return normalcy in children afflicted with rare genetic disorders.
How much is that worth?
Nothing?
What is the return on investment for these valuable agencies?
Treatments for rare diseases in children (click here)Researchers used a personalized gene-editing approach to mend a faulty gene in a baby born with a rare, often deadly disorder. This type of gene-editing method could be used to create personalized therapies for treating additional genetic disorders. Other scientists used an experimental small molecule to alleviate CoQ10 deficiency and reverse associated brain damage in a boy with a rare disease caused by particular genetic mutations. Understanding the molecular causes of rare diseases can lead to effective cures and treatments.
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