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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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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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The CEO of a biomedical startup had made headlines this year when he injected himself with an untested herpes vaccine in front of an audience.

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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: 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: Salk Institute Suspends Cancer Scientist Inder Verma

Salk Institute Suspends Cancer Scientist Inder Verma

By Ashley Yeager | April 23, 2018

The prominent researcher has been put on administrative leave pending an investigation into unspecified allegations.

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image: World’s Largest Cell and Gene Therapy Plant Opens

World’s Largest Cell and Gene Therapy Plant Opens

By Catherine Offord | April 11, 2018

Lonza will employ more than 200 full-time staff to work at the Texas-based facility, the company says.

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