Rare Disease in the News

WFAA

5-year-old needs marrow transplant for rare disease
WFAA
His name is Cline, and he has a very rare blood disorder. His family hopes to register thousands of North Texans for the bone marrow registry in an effort to save his life. Cline McMurray, the "boy in the window," should be "a boy in school." "My name ...

and more »

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 ...


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 ...


Belfast Telegraph

Rare disease consultation plan launched at Stormont by Health Minister Jim Wells
Belfast Telegraph
It is crucial that we do so, because without an organisation to bring together and focus the efforts of all those living and working with rare disease Northern Ireland, families and people affected by rare diseases here will be left vulnerable ...


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 ...

and more »

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.

and more »

Girl with rare disease drafted by Hartford basketball team
WDIV Detroit
Reporter: TWO YEARS AGO ZOE WAS DIAGNOSED WITH A DISEASE THAT ATTACKS HER IMMUNE SYSTEM AND BRAIN AFFECTING BALANCE, AND MOTOR SKILLS. BUT IN ORDER TO PLAY FOR U HEART, HEART IS ALL YOU REALLY NEED.

and more »

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 ...


FDA.gov (blog)

The more we know about rare diseases, the more likely we are to find safe and ...
FDA.gov (blog)
Janet Woodcock You may be inclined to think that rare diseases affect only a tiny fraction of the more than 320 million people in our country. That's true about a single rare disease. But there are about 7,000 rare diseases. If you add them all ...


3rd grader raises more than $800000 for rare disease with 'Chocolate Bar' book
Today.com
Video: When his best friend was diagnosed with a glycogen storage disease, third-grader Dylan Siegel decided to do something to help those suffering from the rare disorder. NBC's Kate Snow reports.