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image: Fighting Canavan Disease

Fighting Canavan Disease

By The Scientist Staff | May 1, 2018

Meet the parents seeking to fund research into a rare disease that afflicts their two boys.

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The devices, which could one day treat children with esophageal atresia and short bowel, were recently tested in pigs.

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When the O’Neills learned that their daughter had Sanfilippo syndrome, a devastating rare disease, they created a GoFundMe campaign that raised $2 million in less than a year.

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Crowdfunding can power investigations into diseases that would otherwise receive little attention.

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image: Infographic: Piecing the Cholesterol Puzzle

Infographic: Piecing the Cholesterol Puzzle

By Diana Kwon | May 1, 2018

How a rare disease led to an understanding of the basics of cholesterol regulation.

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image: Infographic: Rare Disease by the Numbers

Infographic: Rare Disease by the Numbers

By The Scientist Staff | May 1, 2018

How rare conditions and research spending on them compare with more common diseases.

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image: Slow March Toward a Canavan Cure

Slow March Toward a Canavan Cure

By Ashley Yeager | May 1, 2018

Two decades after a successful crowdfunding campaign, some clinical trial patients have seen improvements—but there’s still no approved treatment for the disease.

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Though Calliope Joy’s disease is too far progressed to be treatable, her parents have helped other children with metachromatic leukodystrophy get access to an experimental therapy.

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image: Trailblazing Endocrinologist Neena Schwartz Dies

Trailblazing Endocrinologist Neena Schwartz Dies

By Kerry Grens | April 27, 2018

The reproductive biologist uncovered hormones important for fertility cycles.

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image: Worms’ Magnetic Sense Questioned

Worms’ Magnetic Sense Questioned

By Abby Olena | April 25, 2018

Unsuccessful attempts to reproduce the results of a 2015 study reporting that C. elegans orient themselves by Earth’s magnetic field spark debate among researchers.

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