Rare Disease in the News
Rare disease linked to fleas returns to Galveston
At least 20 people have been treated for the disease at UTMB. Infectious disease expert Dr. Lucas Blanton first noticed its comeback in 2012 when a patient displayed a fever and rash. "It looked and smelled like it could be murine typhus. Then months ...
Six-month-old Piedmont baby diagnosed with rare disease, given 2 years to live
An MRI, blood work and spinal tap confirmed she was 1 of 100,000 diagnosed with the rare disease. This degenerative disorder known as Krabbe will continue to weaken her muscles, inhibiting her ability to chew, swallow and breathe, as well as causing ...
Young Titan Rallies Against Rare Disease
MANHATTAN, Kan. (WIBW) - Smiles and squeals fill the Manhattan home where four-year-old Avalyn Larson is playing with her 11-month-old brother, Titan. "He's always been very happy," dad Lee says of his younger child. "He loves his sister. His sister is ...
Parents turn to data after son is diagnosed with ultra-rare disease
Four years ago, Matthew Might, PhD, and his wife, Christina, learned that their son Bertrand was the first person to be diagnosed with ultra-rare genetic disorder called N-Glycanase Disorder. At the 2015 Big Data in Biomedicine conference at Stanford ...
Family celebrates milestone birthday for son with rare disease
GREENWOOD, Ind. (WISH) — A very special birthday for an Indiana boy whose family made national headlines in the fight against an extremely rare disease is Monday. 24-Hour News 8 first shared the story of the Milto family from Greenwood 15 years ago.
Hollywood Filmmaker Races to Save Daughters with Rare Disease
Doctors have identified an estimated 7,000 rare genetic diseases and disorders with patient populations so small that the opportunity to fund research for treatments is just about as uncommon. Approximately 30 million people in the U.S. and 350 million ...
CHMP Backs Three Drugs for Rare Diseases
The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended marketing clearance of three drugs for rare diseases. The committee recommended approval of sebelipase alfa ( Kanuma , Synageva ...
21st Century Cures Bill to Cost $869M for Rare Disease Drug Marketing
Wall Street Journal (blog)
The sweeping Congressional bill, which is designed to jumpstart medical innovation, has a provision that would allow the FDA to grant an extra six months of exclusive marketing rights to a company if an existing drug is approved to treat a rare disease.
21st Century Cures: Rare disease incentives for pharma will cost the ...
The cost of 'Cures': FDA bill's rare disease provisions to cost $869 million
Celebrities and crowdfunding aid Hollywood family devastated by rare disease
In the course of educating themselves about Batten disease, the Gray family started talking with scientists at Sanford Research, a non-profit research organization in South Dakota. The disease is so rare, and attracts so little biomedical research ...
Producer Gordon Gray Hoping For A Facebook Miracle; Race Against Time To ...
Celebs Rally Behind Hollywood Producer for His Daughters' Rare Brain Disease
Gordon Gray Turns to Social Media to Help His Little Girls
Cordlife Harvests Stem Cells Of Newborns In Case Of A Rare Disease
It's like an insurance policy against a rare disease: The stem cells derived from the blood are used in transplants to combat certain types of cancer and blood disorders. But customers are looking beyond current medical uses as advances in stem-cell ...