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Tiffany House was diagnosed with Pompe disease at age 11 after a childhood of multiple respiratory...
United States Government Work
Uploaded on Feb 28, 2013
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Ronald Bartek cofounded the Friedreich’s Ataxia (FA) Research Alliance (FARA) to support families and...
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Ashley Appell is one of over 25 million Americans living with a rare disease. Ashley was born with...
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Dr. Nancy Wexler is the President of the Hereditary Disease Foundation, started by her father Milton...
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The Undiagnosed Diseases Program (UDP) uses the unique combination of medical expertise and special...
Sharon Terry began her journey as a patient advocate setting out to accelerate research...
Dr. Hal Dietz discovered fibrillin-1 as the underlying genetic cause of Marfan syndrome (MFS) in 1991....
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FDA’s Office of Orphan Products Development (OOPD) was established in 1982. Its critical role in...
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The discovery, development, and clinical testing of products whether for rare or common diseases can be...
The years surrounding the passage of the Orphan Drug Act spurred a national awareness of the challenges...
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Representative Henry Waxman was the principal author of the original 1983 Orphan Drug Act (ODA)....
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The National Institutes of Health (NIH) Office of Rare Diseases Research (ORDR) led by Dr. Steve Groft...
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Dr. Robert Campbell developed the Vertical Expandable Prosthetic Titanium Rib (VEPTR), saving the lives...
Jana Monaco has been an advocate for the rare disease community since her son Stephen suffered severe...
Hemin was the first orphan designated drug to receive marketing approval in 1983. It is used to relieve...
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Abbey Meyers pioneered the development and passage of the 1983 Orphan Drug Act, catalyzed by her...
February 27, 2013 – Product Recall – United Pet Group Inc., Voluntarily Withdraws "Ultra Blend...
Uploaded on Feb 27, 2013
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