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Tuesday, May 31, 2016

The Seattle Times

Innovation is needed in the fight against rare diseases
The Seattle Times
The challenges that come with drug development, namely, high research and development (R&D) costs, small patient populations and lack of incentives, while normally daunting, are even more formidable in the world of rare disease. However, history has ...

Tuesday, May 31, 2016

Bidness ETC

FDA approves Ocaliva for rare, chronic liver disease
FDA.gov
PBC is a chronic, or long lasting, disease that causes the small bile ducts in the liver to become inflamed, damaged and ultimately destroyed. This causes bile to remain in the liver, which damages the liver cells over time, and results in cirrhosis ...
Ocaliva approved for rare liver diseaseUPI.com

all 19 news articles »
Tuesday, May 31, 2016

The Pharma Letter (registration)

GSK's Strimvelis approved in Europe to treat very rare disease, ADA-SCID
The Pharma Letter (registration)
The European Commission has approved Strimvelis, the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (severe combined immunodeficiency due to adenosine deaminase deficiency), from UK pharma major ...
GlaxoSmithKline to provide unique child gene therapyBelfast Telegraph
GSK Wins EU Nod For First Ever Gene Therapy For Children: Significant Victory?Bidness ETC
First gene treatment is approved for childrenThe Times (subscription)

all 8 news articles »
Tuesday, May 31, 2016

Albuquerque Journal

Helping kids battle a rare disease
Albuquerque Journal
Those are the moments the Albuquerque couple found out their youngest daughter suffers from a serious rare disease, and then less than five months later that their son has the same disease. Annika, now 3 years old, was hospitalized more than a year ago.

Monday, May 30, 2016

Newser

Rare Disease Can Make You Think You're Dead
Newser
Yet those are among the beliefs plaguing people with Cotard's syndrome, an illness discovered in 1880 that's extremely rare and awful when it strikes, Medical Daily reports. In case you doubt it, there are two studies (here and here) that found a .57 ...

and more »
Monday, May 30, 2016

Daily Herald

Man who was missing last week recently diagnosed with rare disease which may have contributed to suicide
Daily Herald
Tyson Cook, a man who went missing last week and was found deceased on Friday, was recently diagnosed with a rare disease that contributed to his thoughts of suicide, according to a post on the Facebook page, Help find Tyson Cook. The post was ...

Monday, May 30, 2016

WAAY

7-year-old with rare disease takes the internet by storm
WAAY
When you think of most childhood memories, they're usually of fun times playing and enjoying friends. For one Louisville, Kentucky girl, most of her short life has been spent fighting a rare genetic disease called Diamond Blackfan Anemia. DBA is ...

Sunday, May 29, 2016

Brian beats rare disease, Rohini overcomes age to excel in PU exams
Daijiworld.com
Udupi, May 29: “This has been an exceptional feat for us. Two of our students successfully completed their pre-university exams under great hurdles,” said Umesh Naik, principal, Sneha Tutorial College. The two students are Brian D'Souza (17) and Rohini ...

Saturday, May 28, 2016

Live Action News

Baby survives with rare disease despite diagnosis of death
Live Action News
Alyssa Reidhead and her husband Ben we're thrilled when they found out they were pregnant. For the young couple from Idaho, having their first baby was the happiest time of their lives. When their preborn baby was 23 weeks old the Reidhead's went to ...

Saturday, May 28, 2016

WGME

Scarborough little league tournament benefits umpire battling rare disease
WGME
SCARBOROUGH (WGME) -- There are certain things you need for a good baseball game: the coaches, the equipment and the umpires. "If you don't have an umpire, I would hate to see how the game would end up," laughed Jamie Erskine. He's held the title ...