Center for rare diseases // Drug Repurposing

Center for rare diseases

Endeavour Newsletter

Advocacy, Education, and Science to Improve Lives

Individually, rare diseases affect relatively few Americans. Collectively, they cause millions of people and their families to suffer. For many of these diseases, no treatment is available. For some, drugs or other therapies exist, but patients are unable to access them because the cost is too high.

KGI's Center for Rare Disease Therapies addresses these issues. It raises awareness of rare diseases and promotes promising treatments. The center educates students to become biotech industry leaders who understand rare diseases and recognize the need for remedies. It brings together all of the parties who can make a difference and find solutions: patient advocacy groups, pharmaceutical and biotech companies, government agencies, research institutions and nonprofit organization.

The center dedicates its advocacy, education and science to a singular purpose: making more treatments available to improve the lives of people with rare diseases.

Rare Disease DayThe Center's Mission

The mission of the Center for Rare Disease Therapies is to advocate new therapies for rare diseases and to increase awareness of rare diseases through education, science and ethical business practices. The center acts as a nonprofit catalyst that interacts with government agencies like the FDA's Office of Orphan Product Development, patient advocates such as the National Organization for Rare Disorders and the Pharmaceutical Research and Manufacturers Association (PhRMA).

In the long term, the center aims to be a national and global resource for expertise, training and information related to rare diseases.

Endeavour Newsletter

Check out recent CRDT news in the Summer 2013 Endeavour newsletter.


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by Pantheist_1

Confirmed Variant Creutzfeldt-Jakob Disease (variant CJD) Case in Texas
Posted: June 2, 2014
Laboratory tests have confirmed a diagnosis of variant CJD (a fatal brain disorder) in a patient who recently died in Texas. The confirmation was made when laboratory results from an autopsy of the patient’s brain tested positive for variant CJD.
First described in 1996 in the United Kingdom, variant CJD is a rare, degenerative, fatal brain disorder in humans. It is believed to be caused by consumption of products from cows with the disease bovine spongiform encephalopathy (BSE, or "mad cow" disease)

California case raises mad cow suspicions

by _

A California man has died from what tentatively was diagnosed as a rare brain disorder, but his brother said the patient's neurologist is concerned the man was infected with the human version of mad cow disease, if the patient -- Patrick Hicks, 49 -- suffered from the human form of mad cow disease, also known as variant Creutzfeldt Jakob disease, it would be the first domestic-born case of the brain-wasting illness in the United States.
One U.S. case of vCJD previously was detected in a Florida woman, but she is thought to have contracted the illness while in England.Hicks died early Thursday at Reche Canyon Health Care Center in Colton, Calif

Boots, start walkin

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America is facing an alarming epidemic. In 1960, fewer than one in 10 American children were overweight or obese, but today, that number is one in four. Formerly very rare (and very serious) childhood diseases like Type 2 diabetes have become increasingly common.
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Pelosi only got $37,300,000 of earmarks.....

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Drain that swamp nancy!
....Murtha, the defense industry’s darling, has been known throughout his tenure on the defense panel to shell out a large number of earmarks. His biggest earmark in the bill is $23 million for the National Drug Intelligence Center (NDIC), a move that sparked a fierce fight with Rep. Todd Tiahrt (R-Kan.), who earlier this year voted in a private meeting to strip Murtha’s earmark.
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Screening and caring for children with rare disorders.: An article from: The Hastings Center Report
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Stargardt's disease treatment gains orphan designation  — ModernMedicine
Doylestown, PA—Makindus has received orphan designation for its lead product, MI-100, from the FDA's Office of Orphan Products Development for the treatment of Stargardt's disease. “The FDA's approval for our orphan drug designation application for ..

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