A Disease That Makes Children Age Rapidly Gets Closer to a Cure

Jaybee00 · Jul 24, 2024

https://www.nytimes.com/2024/07/24/...e_code=1.9k0.q4xC.Uw4NrJXPahro&smid=url-share

A disease that affects a couple dozen people worldwide gets closer to a cure……if only Francis would have had his lab investigate a disease that affects more people….

Trish · Jul 24, 2024

A cure for an ultrarare disease, progeria, could be on the horizon. The disease speeds up aging in children and dramatically shortens their lives. But, until recently, there was no path toward a highly effective treatment.

Now, a small group of academics and government scientists, including Dr. Francis Collins, the former director of the National Institutes of Health, is working with no expectation of financial gain to halt progeria in its tracks with an innovative gene editing technique.
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SNT Gatchaman · Jul 24, 2024

Jaybee00 said: ↑

f only Francis would have had his lab investigate a disease that affects more people….
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Dr. Collins first became interested in progeria while he was training in medical genetics at Yale University in 1982, almost three decades before he was appointed to lead the N.I.H. One day, he saw a new patient, Meg Casey. She was less than four feet tall, hairless under her wig and wrinkled like an older woman. She was only in her 20s.

She had progeria.
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Dr. Collins was saddened and moved. Almost nothing was known about the disease, which affects just one in 18 to 20 million people. According to the Progeria Research Foundation, there are only 18 known, living patients in the United States. While Ms. Casey and others have survived into their 20s, people with the disease often live to be only 14 or 15 years old, and many of them die from heart attacks or strokes.
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“I thought, ‘Gosh, somebody should work on this,’” Dr. Collins recalled. “Then I went on to other things.”

Nineteen years later, Dr. Collins, who then headed a federal project to map the human genome, was at a party when he was approached by Dr. Scott Berns, a pediatric emergency room physician. He told Dr. Collins that his toddler, Sam, had a fatal disease.

“I don’t know if you’ve heard about it,” Dr. Berns said. “It’s called progeria.”
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A Disease That Makes Children Age Rapidly Gets Closer to a Cure

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