Latest News

Tuesday, September 02, 2014

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 -- $23,800 for a 28-day supply -- is about 3.4 percent higher than ...

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Tuesday, September 02, 2014

Point Clark's Joe Macartney not backing down against 'PSP' disease 0
Owen Sound Sun Times
Provincial swim champ Joe Macartney of Point Clark suffers from progressive supranuclear palsy (PSP), an extremely rare atypical parkinsonism. He and his wife Merri hope to raise awareness about the invisible disability that affects approximately 6 in ...

Tuesday, September 02, 2014

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 ...

Tuesday, September 02, 2014

Youngster who recovered from rare disease meets "thoughtful" John Terry
Your Local 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 ...

Tuesday, September 02, 2014

Youngster who recovered from rare disease meets "thoughtful" John Terry
Your Local 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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Sunday, August 31, 2014

Vienna woman raises funds to battle rare disease
Warren Tribune Chronicle
Vienna woman raises funds to battle rare disease. August 31, 2014. By BOB COUPLAND Tribune Chronicle (bcoupland@tribtoday.com) , Tribune Chronicle. Save |. VIENNA - For the past two years, Vienna resident Nicole Stafford, who has been battling ...

Saturday, August 30, 2014

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 ...

Saturday, August 30, 2014

FOX 29

Man with rare disease needs new heart, lungs
FOX 29
Man with rare disease needs new heart, lungs. Updated: Saturday, August 30 2014, 02:05 PM CDT. Man with rare disease needs new heart, lungs story image. SAN ANTONIO -- Donnie Brown is in need of a new heart and two lungs. In 2012, Brown was ...

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Friday, August 29, 2014

WOAI

Man with rare disease needs a new heart and two lungs
WOAI
More Top Stories. Family Speaks Out For First Time After Son Killed From Industrial Accident Injuries. BREAKING NEWS: AMBER ALERT issued after 3-year-old boy abducted. Man with rare disease needs a new heart and two lungs. Two in custody in murder ...

Thursday, August 28, 2014

Bioscience Technology

Penn Medicine Study: Attacking a Rare Disease at its Source With Gene Therapy
Penn: Office of University Communications
Treating the rare disease MPS I is a challenge. MPS I, caused by the deficiency of a key enzyme called IDUA, eventually leads to the abnormal accumulation of certain molecules and cell death. The two main treatments for MPS I are bone marrow ...
Attacking a Rare Disease at its Source with Gene TherapyBioscience Technology

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