MS. JOHNSON: Hello, and welcome to Post Live. I’m Akilah Johnson, a national health reporter focused on health disparities here at The Washington Post. And today we have two segments on rare diseases.
Rare neurological diseases offer unusually clear models for testing earlier diagnosis, precision treatment and long-term ...
Montez, Medscape’s Rare Disease Education Lead recently spoke with Dr Lucy McKay, CEO, and Megan Pullein, Strategic Operations Officer from the UK-based charity Medics for Rare Disease (MfRD). We ...
Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases collectively impact millions of Americans, creating a healthcare system that’s ...
GEORGETOWN COUNTY, SC (WMBF) - A Georgetown County teenager is preparing to head to Boston, Massachusetts, for a rare surgery to help treat two rare diseases. “He’s a true example to me,” mother ...
The cause of rare diseases is increasingly being detected through genome sequencing, which involves reading the entire human DNA by first breaking it into small pieces—short reads. Christian Gilissen, ...
Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...
Rare Disease Day raises awareness for the thousands of rare diseases affecting millions. Advancements in technology, such as gene therapy, offer hope for potential cures for rare diseases in the ...
Affected populations: POLG-related diseases are a group of rare, incurable conditions caused by inherited gene mutations. These disease-causing mutations are carried by up to 2% of people of Northern ...
While Novo Nordisk has been operating in rare disease for some 45 years, the field has often taken a back seat to the Danish drugmaker’s bread-and-butter business developing treatments for diabetes ...
The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive advocate for the development of rare-disease drugs. When I joined the FDA to be ...