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Friday, October 31, 2014

State Notebook: PJP's Tyler Nugent keeps football dreams alive despite rare ...
The Sentinel
“I'm always going to have it,” he said of the disorder. “I have to live with it. I haven't been able to eat a full meal or an all you can eat buffet in two years. I drink a lot of nutritional drinks and am back to eating sone regular foods like ...

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Thursday, October 30, 2014

KHOU

5-year-old needs marrow transplant for rare disease
KHOU
FORT WORTH, Texas - There's a f5-year-old boy in Fort Worth who needs your help. His name is Cline, and he has a very rare blood disorder. His family. Loading… Post to Facebook. 5-year-old needs marrow transplant for rare disease FORT WORTH, Texas ...

Wednesday, October 29, 2014

WFAA

5-year-old needs marrow transplant for rare disease
WFAA
5-year-old needs marrow transplant for rare disease. A boy with a rare blood disorder needs to find a match for a bone-marrow transplant. News 8's David Schechter has more. David Schechter, WFAA 6:25 a.m. CDT October 30, 2014 ...

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Wednesday, October 29, 2014

KENS 5 TV

Cousin of SA woman afflicted by rare disease: I'm 'starving for a cure'
KENS 5 TV
SAN ANTONIO -- One San Antonio resident said she will be starving herself outside of a local restaurant for two days, all in the hopes to raise money for a rare disease. Erika Pompa said she set up camp on the sidewalk outside of Tost Bistro Fine ...

Wednesday, October 29, 2014

Globalnews.ca

Community of Cardston rallies around boy with rare disease
Globalnews.ca
Zayden Eagle Plume just celebrated his first birthday in the Foothills hospital in Calgary. In fact, the young Cardston boy has not been able to leave since he was born. Eagle Plume has a rare and fatal disease called urea cycle disorder. “What it is ...

Tuesday, October 28, 2014

Belfast Telegraph

Rare disease consultation plan launched at Stormont by Health Minister Jim Wells
Belfast Telegraph
The draft plan - which sets out a six-year framework - was launched at Stormont by the Northern Ireland Rare Disease Partnership (NIRDP) and Health Minister Jim Wells. Around 100,000 people in Northern Ireland are impacted by rare diseases, according ...

Tuesday, October 28, 2014

Cantech Letter

Prometic Plans to Move Quickly With Rare Disease Candidate
Cantech Letter
This remarkably quick path to a potential FDA approval decision on plasminogen is reflective of the small number of patients who have hypoplasminogenemia. Prometic received orphan drug designation for the use of plasminogen in this rare disease in 2013.

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Tuesday, October 28, 2014

WJHL

Johnson City girl with rare disease honored with butterfly release
WJHL
According to Bailey's Father Ronnie bailey, "Children who have Epidermolysis Bullosa are known as "butterfly children" because their skin is as fragile as a butterflies wings...so we do this to create awareness just specifically for the condition ...

Tuesday, October 28, 2014

A Closer Looker at a Big Gift for Rare Diseases to Notre Dame
Inside Philanthropy
Simply put: Sometimes it's personal, and sometimes it just ain't. The latest big gift the University of Notre Dame received to support its Center for Rare and Neglected Diseases illustrates exactly that. We know of plenty of wealthy donors giving to ...

Tuesday, October 28, 2014

New York Times (blog)

When the Diagnosis Is Rare, Parents May Know More Than Professionals
New York Times (blog)
That is when David and Glenda began their foray into the medical field — not as doctors, but as experts in the rare and fatal disease that Katherine may be suffering from: Infantile Neuroaxonal Dystrophy (INAD). Nowadays, David and Glenda's ...

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