The Rarest of All Diseases Are Becoming Treatable

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In February, a six-month-old baby named KJ Muldoon became the first person ever to receive a CRISPR gene-editing treatment customized specifically for his unique genetic mutation, a milestone that researchers say marks a turning point in how medicine might approach the thousands of rare diseases that collectively affect 30 million Americans. Muldoon was born with a type of urea-cycle disorder that gives patients roughly a 50% chance of surviving infancy and typically…



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The Rarest of All Diseases Are Becoming Treatable


In February, a six-month-old baby named KJ Muldoon became the first person ever to receive a CRISPR gene-editing treatment customized specifically for his unique genetic mutation, a milestone that researchers say marks a turning point in how medicine might approach the thousands of rare diseases that collectively affect 30 million Americans. Muldoon was born with a type of urea-cycle disorder that gives patients roughly a 50% chance of surviving infancy and typically…



Source link

Disclaimer

We strive to uphold the highest ethical standards in all of our reporting and coverage. We StartupNews.fyi want to be transparent with our readers about any potential conflicts of interest that may arise in our work. It’s possible that some of the investors we feature may have connections to other businesses, including competitors or companies we write about. However, we want to assure our readers that this will not have any impact on the integrity or impartiality of our reporting. We are committed to delivering accurate, unbiased news and information to our audience, and we will continue to uphold our ethics and principles in all of our work. Thank you for your trust and support.

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