Rare Disease in the News

CBS Local

Young boy with rare disease gets to fly like Iron Man
An 8-year-old boy with a rare disease was given the chance to fly like his hero Iron Man in Colorado earlier this week. ironmankid. Max Vertin, via KCNC. Max Vertin suffers from Duchenne muscular dystrophy, which slowly robs his muscles of their abilities.
8-Year-Old With Rare Disease Becomes 'Iron Man' For A DayCBS Local
Eight-year-old boy with rare degenerative disease fulfils wish to fly like ...Metro
8-yr.-old boy with rare disease lives out his dream of flyingWNCT
The Republic -Daily Mail -Charlotte Observer
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David, Goliath Team Up in Battle to Cure Rare Diseases
Fox Business
When Kathy Giusti was diagnosed with a rare and fatal blood cancer nearly 20 years ago, there wasn't a single drug in the pipeline to help her fight her grim prognosis, and she was expected to die within three years. That is until the then-new mother ...

VCs gamble $96M on rare disease drug, biosimilar play and new drug for AMD
Making up for an anemic first quarter, a group of VCs operating on both sides of the Atlantic has triggered a string of new rounds totaling $96 million to back three biotech upstarts. The rare-disease biotech Wilson Therapeutics in Stockholm led the ...

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Rare Disease May Hold Secret to Stopping Viruses
A brother and sister with an extremely rare genetic condition have a stunning ability to fight off viruses — an ability that could lead to a new family of antiviral drugs. Their bodies stop viruses from building a protective shell, which leaves ...

Winnipeggers help support families facing rare disease
Batten disease is a rare inherited disorder of the nervous system that develops when a child has two defective genes. There is no cure and it is always fatal. It is unusual for children with Batten disease to live beyond their teens. The disease only ...

KETV Omaha

South Carolina family of 4-year-old girl with rare disease raises $400000 for cure
New York Daily News
The family of Eliza O'Neill has garnered about $160,000 on its GoFundMe.com site alone since releasing the video, which explains Eliza's story and the extremely rare disease called Sanfilippo syndrome, with which she was diagnosed in July. Eliza's ...
Parents raise funds for daughter's rare terminal diseaseKETV Omaha

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Here's how the government pushes companies to treat kids and rare diseases
SFGate (blog)
The reason BioMarin is in the business of rare diseases in the first place goes back to 1983, when the nation passed the Orphan Drug Act. Before then, few companies made drugs for orphan diseases because there were relatively few patients who would buy ...

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Research Gives New Insights Into Rare Disease Of The Inner Ear
A new study has shed light on the factors likely to lead to the development of a rare condition affecting the inner ear. In the most comprehensive study of Ménière's Disease to date, researchers at the University of Exeter Medical School have been able ...
Study sheds light on factors that lead to development of rare condition ...News-Medical.net

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Woman's quest could help cure rare disorder
ST. LOUIS (AP) — When Mary Kaye Richter's son was diagnosed, she could only find a few paragraphs about hypohidrotic ectodermal dysplasia, which inhibits the growth of teeth, hair and sweat glands. To learn more, she converted a storage room of a ...

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Genetic research leads to treatment for rare disease
Greenville News
A new treatment that could help children born with a rare genetic condition has been developed based on a two-decades-old discovery by a researcher now at the Greenwood Genetic Center. jonholloway-77.jpg. Dr. Anand Srivastava (Photo: jon holloway ).