Rare Disease in the News

His body contorted, Warren man's video may boost awareness about rare disease
Detroit Free Press
Twisting its victims into jarring and often painful contortions, a little-known disease would get its own awareness month if a Warren man gets his way. And Mike Delise is using a video of his friend writhing uncontrollably on a carpeted floor to make ...

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New Genzyme pill will cost patients $310250 a year
Boston Globe
Cambridge biotech Genzyme has priced its new pill to treat Gaucher disease at $310,250 a year for the small population of US patients suffering from the rare genetic disorder. The price — listed as $23,800 for a 28-day supply — is slightly higher ...

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Shenzhen parents seek medical help for baby suffering rare disease
Shanghaiist
Finding help from doctors has exhausted the family's savings, and the couple still can't find the cause of Yuanyuan's rare disease or proper treatment to help. The couple is now reaching out through media to seek medical experts who could give a formal ...


The Guardian

Trends in Rare Diseases – or why the ice bucket challenge is a cold dash of ...
The Guardian
To be fair, there are some rare diseases mentioned in some of the existing journals. Trends in Parasitology covers diseases that are not rare at all, but are still not trendy – anyone you know affected by schistosomiasis? Nonetheless, literature on ...


Bidness Etc

Amicus Releases Positive Study Results On Rare Disease Drug
Forbes
Amicus Therapeutics FOLD -1.1% is this morning releasing data on its pill, migalastat, for Fabry disease, a rare genetic disorder in which fat builds up in the body, causing pain, hearing loss, clouded eyes, and, eventually kidney failure, stroke, and ...
Amicus sets sights on approval as rare disease pill clears Phase IIIFierceBiotech

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Rare disease could come right from your medicine cabinet
kdvr.com
The disease is called “Stevens Johnson Syndrome” or SJS. It's an extremely rare disease that is most commonly caused by antibiotics like penicillin or Bactrim. It impacts less than 1 percent of the people who use them each year. The family of 24-year ...
The rare disease that could come from your medicine cabinetfox2now.com
Family: Rare disease claims daughter's life after she took medicine for bug bitefox8.com
New mom dies from rare disease after taking medicine for a bug biteFOX43.com

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MIT Technology Review

Ice Bucket Challenge highlights how rare diseases are neglected
ModernHealthcare.com
ALS, called Lou Gehrig's disease because it took the life of the legendary New York Yankees first baseman, is a rare disease. About 1 in 55,000 Americans, or 5,600 people, are diagnosed annually with the disorder, a progressive deterioration of the ...
How ALS Could Benefit from a Flood of New FundingMIT Technology Review
Why the Ice Bucket Challenge is bad for youMacleans.ca
Chinese Tycoons, Stars Take up Ice Bucket Challenge for ALSCaixin Media
South China Morning Post (subscription) -Washington Post (blog) -CCTV
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Bill to screen newborns for rare disease heads to governor
Bakersfield Now
The 14-year-old high school freshman suffers from a rare genetic disorder called Adrenoleukodystrophy, or ALD for short. ALD affects mostly boys that causes seizures and can rapidly lead to a vegetative state and death. In Jeremy's case, it has robbed ...


Youngster who recovered from rare disease meets "thoughtful" John Terry
Epsom Guardian
A young Chelsea fan who has just recovered from a rare disease which left her in a coma for nine months said meeting John Terry “marks the end” of her difficult journey. Arabella Webster, 20, from Epsom, was diagnosed with a rare auto immune disease ...

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Bioscience Technology

Attacking a Rare Disease at its Source with Gene Therapy
Bioscience Technology
This family of diseases comprises about 50 rare inherited disorders marked by defects in the lysosomes, compartments within cells filled with enzymes to digest large molecules. If one of these enzymes is mutated, molecules that would normally be ...
Penn Medicine Study: Attacking a Rare Disease at its Source With Gene TherapyPenn: Office of University Communications

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