Rare Disease in the News
Rare disease makes Lincoln baby almost untouchable
The Kinkles are sharing their story in hopes of raising awareness about the disease with the hope that it will lead to greater funding for research of a cure. "It's so rare there's not a whole lot of awareness and therefore not a whole lot of money ...
Baby's Rare Disease Makes Hugging Too Dangerous
Rare disease makes baby girl almost untouchable
Baby's rare disease means parents cannot hug her
Alexion's Rare Disease Model Analyzed
Alexion Pharmaceuticals (NASDAQ:ALXN) is best known for its Soliris therapy for rare diseases. It represents a new biotechnology model, in which investing in finding therapies for rare diseases is highly rewarding to stockholders. Each patient is ...
New hope for rare disease drug development
Using combinations of well-known approved drugs has for the first time been shown to be potentially safe in treating a rare disease, according to the results of a clinical trial published in the open access Orphanet Journal of Rare Diseases. The study ...
More than $100G donated to nonprofit founded for Marisa Carney, Bayport girl ...
Marisa Carney, 8, has been living on a portable ventilator since 2011. She is the only person on Long Island to be diagnosed with ROHHAD, an acronym for Rapid-onset Obesity with Hypothalamic Dysfunction, Hypoventilation and Autonomic Dysregulation.
ABC News (blog)
8-Year-Old Raises More Than $1 Million to Help Cure His Friend's Rare Disease
Some best friends will do anything for each other – even if it means raising more than $1 million. In 2012, when Dylan Siegel, then 6, found out his best pal, Jonah Pournazarian, had a rare liver disorder called glycogen storage disease type 1B, he ...
Eight-year-old boy's "Chocolate Bar" books raises $1 million to help research ...
Boy's 'Chocolate Bar' Book Nets $1M for Rare Liver Disorder Research
8-Year-Old Boy Raises $1 Million For Research For Best Friend's Rare Disease
Boston Business Journal (blog)
Former Genzyme exec Vivaldi heads to Philadephia rare disease biotech
Boston Business Journal (blog)
The move back to rare diseases fits in with Vivaldi's background as a pioneer in rare diseases. He was the first physician in his native country of Brazil to treat the rare disease, Gaucher, when Genzyme first came out with an enzyme replacement ...
Spark Therapeutics Appoints Rare Disease Pioneer Dr. Rogerio Vivaldi as Chief ...
Surviving the Rare Disease Roller Coaster
U.S. News & World Report
And indeed, that's a common misdiagnosis for the devastating disease Eliza actually had: Sanfilippo syndrome, a rare genetic metabolic disorder that gets progressively worse in children after age 5 and typically causes their death when they're in their ...
Family raises $1.8M for SC girl with rare disease - CBS 3 Springfield - WSHM
Lance to chair Congressional Rare Disease Caucus again
The Ripon Advance
Rep. Leonard Lance (R-N.J.) will continue to serve as the Republican Chair of the Congressional Rare Disease Caucus during the 114th Congress, which begins in January. The caucus builds support for legislation that helps constituents with rare diseases.
Rare disease public meetings will help shape the future of healthcare in ...
A spokeswoman for NIRDP said: "Whether you are a patient, a carer, a health or social care professional, or someone who works with people living with a rare disease in education, employment or any other aspect of life, these meetings give you the ...