Latest News

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
In 2012, Brown was diagnosed with Scleraderma, an auto immune disease. "We didn't know what it was", said Brown. The disease has progressed and is now effecting his heart and lungs. "It's been very difficult, life changing, I mean there are really no ...

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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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Thursday, August 28, 2014

Girl battles rare disorder, focuses on helping others
WDIV Detroit
SHE IS A CHILD WITH A RARE DISORDER. SHE HAS BEEN THROUGH MORE THAN MOST ADULTS HAVE. AND ROAD AHEAD WILL BE A VERY BUMPY LONG ROAD FOR HER. BUT ELANA'S FOCUS IS ON OTHERS NOT HERSELF. IT MAKES ME ...

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Thursday, August 28, 2014

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 ...

Thursday, August 28, 2014

Edinburgh Evening News

Bucket list for step-brothers with rare disease
Edinburgh Evening News
Both treasure their iPads, share a fascination for the game Angry Birds and – despite odds of “more than a million to one” – they each suffer from a rare muscle wasting disease. The illness, called Duchenne Muscular Dystrophy (DMD), means the ...

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Thursday, August 28, 2014

Rappler

Angara accepts ice bucket challenge, files rare disease bill
Sun.Star
ASIDE from taking on the ice bucket challenge on Thursday, Senator Edgardo Sonny Angara filed a Rare Diseases Bill, which aims to provide patients with rare diseases and their families better access to adequate medical care, health information, and ...
Angara does ice bucket challenge, files bill for coverage on rare diseasesInquirer.net
Angara bill to help patients with ALS, rare diseasesRappler
WATCH: Angara does ice bucket challengeABS CBN News

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Thursday, August 28, 2014

Rappler

Angara does ice bucket challenge, files bill for coverage on rare diseases
Inquirer.net
Aside from taking on the challenge, Angara has also filed the Rare Diseases Bill, which seeks to provide patients with rare diseases, including the amyotrophic lateral sclerosis or ALS, and their families better access to adequate medical care, health ...
Angara accepts ice bucket challenge, files rare disease billSun.Star
Angara bill to help patients with ALS, rare diseasesRappler
WATCH: Angara does ice bucket challengeABS CBN News
GMA News
all 8 news articles »
Wednesday, August 27, 2014

Girl battles rare disorder, focuses on helping others
WDIV Detroit
SHE IS A CHILD WITH A RARE DISORDER. SHE HAS BEEN THROUGH MORE THAN MOST ADULTS HAVE. AND ROAD AHEAD WILL BE A VERY BUMPY LONG ROAD FOR HER. BUT ELANA'S FOCUS IS ON OTHERS NOT HERSELF. IT MAKES ME ...

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