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image: Positive Trial Results for Experimental DMD Gene Therapy

Positive Trial Results for Experimental DMD Gene Therapy

By Catherine Offord | June 20, 2018

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.

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Doctors treated the fetus, who has alpha thalassemia major, with cells from her mother’s bone marrow.

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Biotechs have developed enzyme replacements and genetically modified probiotics to treat patients with the rare metabolic disorder.

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As the first personalized cell and gene therapies are approved from small clinical trials, researchers propose the creation of publicly accessible databases to pull together real-world results.  

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image: Ali’s Journey

Ali’s Journey

By The Scientist Staff | May 1, 2018

Ali Guthy, the daughter of cosmetics entrepreneur Victoria Jackson, discusses NMO, the rare autoimmune disease she suffers from.

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image: Among the Amish, c. 1960s

Among the Amish, c. 1960s

By Diana Kwon | May 1, 2018

Victor McKusick’s pioneering investigations provided insight into hereditary disorders.

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image: Book Excerpt from <em>The Power of Rare</em>

Book Excerpt from The Power of Rare

By Victoria Jackson and Michael Yeaman | May 1, 2018

In chapter 4, “Building a Cure Machine,” author Victoria Jackson reveals the challenges in launching a foundation focused on funding research on a rare disease.

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image: Bullets and Ballots

Bullets and Ballots

By Bob Grant | May 1, 2018

Researching the rare, but all-too-common, disease of gun violence in America will take a concerted political effort.

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image: Caught on Camera

Caught on Camera

By The Scientist Staff | May 1, 2018

Selected rare-disease Images of the Day from the-scientist.com

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image: Copper Connections

Copper Connections

By Jim Daley | May 1, 2018

Researchers initially set out to investigate the relationship between copper homeostasis and the rare Menkes disease, but they also found links to Parkinson’s.

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