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image: Saving Jon

Saving Jon

By | October 1, 2016

Meet the researcher/entrepreneur who started a nonprofit that seeks to solve the science behind a rare disease that threatens the life of her younger brother.

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image: The Challenges of Rare-Disease Research

The Challenges of Rare-Disease Research

By | September 1, 2016

With few resources and hesitant investors, basic scientists must rely on clinicians, patient advocates, and their own keen eye for biological connections.

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image: The Genetic Components of Rare Diseases

The Genetic Components of Rare Diseases

By | July 1, 2016

Techniques for determining which genes or genetic variants are truly detrimental

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image: Another Fatal-Disease Drug in Limbo

Another Fatal-Disease Drug in Limbo

By | April 26, 2016

A federal advisory panel votes against Sarepta Therapeutics’s treatment for Duchenne muscular dystrophy.

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image: $280 Million Boost for Disease Genomics

$280 Million Boost for Disease Genomics

By | January 18, 2016

The genomics arm of the National Institutes of Health has pledged a total of $280 million for research into the genetic bases of disease.

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image: Shire to Buy Baxalta for $32 Billion

Shire to Buy Baxalta for $32 Billion

By | January 12, 2016

The Dublin-based pharma giant is set to acquire Baxalta, an Illinois-based Baxter spinoff, expanding its rare disease drug portfolio.

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image: Shire Buys Rare Disease Drug Developer

Shire Buys Rare Disease Drug Developer

By | November 2, 2015

The Irish drugmaker is set to acquire the Massachusetts-based Dyax in a $5.9 billion deal.

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image: Spinoff Company to Develop Rare Disease Drugs

Spinoff Company to Develop Rare Disease Drugs

By | January 7, 2015

Maryland-based Vtesse will work with the US National Institutes of Health to advance treatments for Niemann-Pick Type C disease and other lysosomal storage disorders.

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image: Uncommonly Rare

Uncommonly Rare

By | November 1, 2014

How one of the rarest neurodegenerative diseases could lend insight into ubiquitous neuroprotective processes

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image: Mosaic Mutations

Mosaic Mutations

By | July 31, 2014

Some genetic abnormalities that appear to have sprung up independently in children are in fact present in a portion of their parents’ cells.

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