Rare Disease in the News

WPTV.com

Rare disease makes Lincoln baby almost untouchable
KCRA Sacramento
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 DangerousNBCNews.com
Rare disease makes baby girl almost untouchableNBC2 News
Baby's rare disease means parents cannot hug her9news.com.au
WPTV.com -Kansas First News -Science World Report
all 16 news articles »

Alexion's Rare Disease Model Analyzed
Seeking Alpha
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 ...

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New hope for rare disease drug development
Medical Xpress
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 ...

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Man suffers rare disease
WJXT Jacksonville
Man suffers rare disease. Published On: Dec 17 2014 10:37:48 PM EST Updated On: Dec 17 2014 10:55:34 PM EST. Small Text; Medium Text; Large Text. Print; Email. Tweet. Steve Austin was diagnosed with a rare blood mutation called "Jak-2" and it ...

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More than $100G donated to nonprofit founded for Marisa Carney, Bayport girl ...
Newsday
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.

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ABC News (blog)

8-Year-Old Raises More Than $1 Million to Help Cure His Friend's Rare Disease
TIME
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 ...abc7news.com
Boy's 'Chocolate Bar' Book Nets $1M for Rare Liver Disorder ResearchABC News (blog)
8-Year-Old Boy Raises $1 Million For Research For Best Friend's Rare DiseaseWRIC
Islamabad Globe
all 38 news articles »

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 ...The Providence Journal

all 5 news articles »

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 - WSHMWSHM-TV

all 9 news articles »

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.


Belfast Telegraph

Rare disease public meetings will help shape the future of healthcare in ...
Belfast Telegraph
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 ...