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Orphan Drugs for Rare Diseases

Find information here about drugs developed to treat rare diseases and disorders.

Top Orphan Drugs Approved in 2008
Marketing approval for orphan drugs, specialty drugs created to treat rare diseases, is always exciting. In 2008, the U.S. Food and Drug Administration (FDA) approved several of these drugs that were particularly important.

What is an Orphan Drug?
Article describes what an orphan drug is, and how the United States and European Union goverments support orphan drug research and development.

Orphan Drugs Approved by the FDA for Marketing in 2007
Information about the orphan drugs approved by the U.S. Food and Drug Administration (FDA) for marketing in 2007, including trade and generic names, use, date of approval, and manufacturer.

Orphan Drugs Approved by the FDA for Marketing in 2006
Article lists the orphan drugs approved by the FDA for marketing in the US in 2006, their uses, and their manufacturers.

Two Orphan Drugs Approved
Treatments are now available in the U.S. for two rare diseases: Fabrazyme, for Fabry disease, and Aldurazyme, for MPS I. Both are storage disorders with devastating effects in their severest forms.

First Hurler Drug in Clinical Trials
A critical body enzyme is deficient in MPS I, a genetic disorder. Remedies for both its severe (Hurler) and mild (Scheie) syndromes have been elusive until now: the first specific drug treatment is producing promising results in clinical trials. From the About.com Guide to Rare/Orphan Diseases.

The Return of Thalidomide
Feature article discusses thalidomide's present uses to treat rare diseases and its dark history of birth defects.

Trends in Orphan Drug Research for 2006
Article reviews trends in orphan drug research and the biotechnology industry in 2005 and 2006.

Orphan Drugs Approved in 2005
These orphan drugs (specialty drugs to treat rare disorders or conditions) were approved by the U.S. Food and Drug Administration (FDA) for marketing in the U.S. in 2005. Internet links for more information are provided where available.

Trends and Issues in 2005 for Orphan Drug Research
Article reviews trends and issues regarding orphan drug research and the biotechnology industry.

Orphan Drugs Approved in 2004
These orphan drugs (specialty drugs to treat rare disorders or conditions) were approved by the U.S. Food and Drug Administration (FDA) for marketing in the U.S. in 2004. Internet links for more information are provided where available.

Orphan Drugs Designated in 2003
These drugs and treatments were granted orphan drug status by the U.S. Food and Drug Administration in 2003. With each drug is listed what condition it is designed to treat, and which company is sponsoring it. Links to more information are provided where available.

Orphan Drugs Approved in 2003
These orphan drugs (specialty drugs to treat rare disorders or conditions) were approved by the U.S. Food and Drug Administration (FDA) for marketing in the U.S. in 2003. Internet links for more information are provided where available.

Orphan Drugs 2002
Here is a list of the orphan drugs--medications and treatments for rare diseases--approved in 2002 for marketing, with links to more information about them and the conditions they were designed to treat.

FDA Puts Hold on Gene Therapy Studies
The U.S. FDA temporarily halted gene therapy studies after two children who received this experimental therapy for a rare disorder developed cancer. The studies will most likely resume, but with new cautions.

Vaccines for Rare Diseases
What is an orphan vaccine? Why isn't this method of prevention or treatment being developed? Learn how you can network with other concerned individuals, participate in clinical trials, and advocate for more research and development.

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