News tagged with rare disease

Rapamune approved for rare lung disease

(HealthDay)—Rapamune (sirolimus) has been approved by the U.S. Food and Drug Administration to treat a rare, progressive lung disease that mostly affects women of childbearing age.

May 29, 2015
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Stem cell disease model clarifies bone cancer trigger

Using induced pluripotent stem cells (iPSCs), a team led by Mount Sinai researchers has gained new insight into genetic changes that may turn a well known anti-cancer signaling gene into a driver of risk for bone cancers, ...

Apr 09, 2015
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Crowdfunding helps solve rare disease mystery

Rare diseases—those that affect fewer than one in 200,000 people—are often identified early in life. Some 30 percent of children afflicted by these "orphan diseases" do not live to see their fifth birthday. ...

Feb 10, 2015
popularity 26 comments 0

FDA drug approvals reached 18-year high in 2014

The Food and Drug Administration approved 41 first-of-a-kind drugs in 2014, including a record number of medicines for rare diseases, pushing the agency's annual tally of drug approvals to its highest level in 18 years.

Jan 02, 2015
popularity 12 comments 0