Rare Disease in the News

The Sun Chronicle

North Attleboro woman trying to gain attention for Rare Disease Day ...
The Sun Chronicle
NORTH ATTLEBORO — A local woman who started an advocacy organization after struggling with her own rare disease wants others to learn more about the ...

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The Sun Herald

Two 'unstoppable' mothers honored for efforts to save their children
The Sun Herald
Willow Cannan is living with an extremely rare genetic disease called Multiple Sulfatase Deficiency. It's such a rare disease that there is no treatment available for it. Children that are diagnosed with MSD are not expected to live past the age of 10 ...


wivb.com

Local 2-year-old running out of time with rare disease
wivb.com
WILLIAMSVILLE, N.Y. (WIVB) — A local 2-year-old girl is dealing with a rare disease. Doctors told the family there's not enough research and they're still trying to find the best treatment for her. The family says her health is getting worse the ...


Newron Supports Global Rare Disease Day® 2017 and Rett Syndrome Studies
Yahoo Finance
Newron Pharmaceuticals S.p.A. (“Newron”), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central nervous system (CNS) and pain, today announced its support of this year's Rare Disease Day® ...

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CNBC

How a start-up has found a dozen treatments for rare diseases using robots
CNBC
Cerebral cavernous malformation isn't the kind of disease the average person is familiar with. But for the 0.5 percent of the population afflicted with the genetic affliction — which causes blood vessels in the brain to grow irregularly — every day ...


Living with the rare disease, dystonia: 'It's like charley horses all over'
Fox17
Wirts has dystonia, a rare neurological movement disorder. It's the third most common movement disorder behind Parkinson's disease and essential tremor, but it is still considered a rare disease that affects only 300,000 people in all of North America.


EverythingLubbock.com

Guthrie Teen With Rare Disease Receives Surprise Award
EverythingLubbock.com
LUBBOCK, TX - Shaylee Boger was diagnosed with Pompe disease, an inherited disorder that disables the heart and skeletal muscles. Today she was honored by the Sanofi Genzyme Organization with the Torch award; which recognizes individuals who ...


Sacramento Bee

Orphan drugs offer hope for treating rare diseases, despite high ...
Sacramento Bee
So-called orphan drugs designed to treat rare diseases offer patients some hope of improvement, despite the high price tag. One boy in Turlock who suffers from ...
Outrageous price, approval a concern for drug to treat deadliest ...CTV News

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Bloomberg

Tough U.K. Line on Rare-Disease Drugs Stirs Concern After Brexit ...
Bloomberg
A tougher stance in the U.K. on funding expensive medicines for rare diseases is raising questions about the government's pledges to support a life-sciences ...

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Korean Startup 3billion Focuses on Rare Disease Community With DTC Exome Screening Test
GenomeWeb
NEW YORK (GenomeWeb) – Korean startup 3billion is getting ready to offer a direct-to-consumer genomic rare disease screening test to patients in the US who are looking for a molecular diagnosis and haven't been able to obtain one otherwise.