Rare Disease in the News

aTyr Pharma Appoints Rare Disease Pioneer Mark Goldberg, MD, to Board of ...
MarketWatch
He has an impressive track record in developing medicines for rare diseases and has successfully navigated the unique commercial and regulatory challenges associated with delivering these new treatments to patients in need," said John Mendlein, Ph.D., ...
aTyr's Resolaris granted FDA Orphan Drug Designation for treatment of FSHDNews-Medical.net

all 4 news articles »

16-month-old St. Cloud boy battling two rare diseases
News 13 Orlando
Dylan Austin is just 16 months old, but he's already fighting two diseases — including a rare bacterial infection in both his blood and bones. His parents, who live in St. Cloud, said they have received prayers from all over the word in support of ...

and more »

FOX31 Denver

Colorado boy with rare disease inspires others around the world to be ...
FOX31 Denver
DENVER — For years they fought their fight alone. That was until an 11-year-old from Denver decided to look outside his pain for others who are battling the same disease. Patrick Boland found companions in Colorado. Their bond is helping to fight a ...


New gene therapy success in a rare disease of the immune system
Science Daily
For very rare diseases such as WAS, multicenter clinical trials are the only effective way of proving the safety and efficacy of gene therapy and having it rapidly approuved and made available to all patients. We are following the same approach for ...


The Ulster Herald

Family speak of schoolboy's rare disease diagnosis
The Ulster Herald
AGED just 11, Aghyaran schoolboy Damian McHugh is coping with a lot more than most children his age. Last year the local schoolboy was diagnosed with the extremely rare Ollier's disease. Affecting just one in 100,000 people, the non-hereditary disorder ...


Fox News

Belgium, Netherlands plan joint purchase of rare disease drugs
Fox News
The issue of expensive medicines for rare diseases sparked a debate in the Belgian media two years ago, when the health ministry refused to reimburse the treatment for a seven-year old boy suffering from a rare kidney disease, which cost 9,000 euros ...

and more »

District 58 schools raise money to fight rare diseases
Suburban Life Publications
The Kingsley Student Council hosted a "Dance Off Disease Day," raising more than $700 for the Global Genes Fund. The Lester School Student Council sponsored a penny harvest to collect money for the Global Genes Project and teachers could pay a dollar ...

and more »

Peoria Journal Star

In the Spotlight: When rare disease strikes, it doesn't seem rare
Peoria Journal Star
The March 26 Journal Star included an article entitled “Science, patients driving rare disease drug research surge.” As the husband and caregiver of a woman with the rare and terminal disease Multiple System Atrophy (MSA), the article hit home. The ...


Medical Xpress

Scientists discover genetic cause of deadly rare disease 'dyskeratosis congenita'
Medical Xpress
Published in the Journal of Clinical Investigation, the researchers used the latest genetic sequencing methods to study 31 children with the disease and discovered a defect in the biological process known as deadenylation. This is the routine breakdown ...


Dream comes true for Vt. boy battling rare disease
WCAX
This guy has been smiling the whole time," the boy's father said. Cohen was born with VACTERL association, a rare condition which prevents his organs from functioning. He has undergone 24 surgeries so far. "It's incredibly emotional," mom Amy Cohen said.

and more »