Orphan drugs, for those of you who don’t know, are those designated especially for rare diseases. They respond to a public health need, but not necessarily an economic need by…
Nope, we're not joking! US SWAT teams (Special Weapons And Tactics, military weapons-equipped law enforcement teams) have been asked to step in to help with backlogged FDA applications to get experimental treatments designated as orphan…
As most readers of this site are probably aware, a “rare disease” is classified in the United States as one that affects fewer than 200,000 people. It’s estimated there are…
Bronchiectasis is a condition which damages the airways leading in and out of the lungs, stripping them of their mucus-clearing capabilities. Common variable immune deficiency (or CVID) is an immune…
For many people suffering from rare diseases, gene therapy offers the hope for relief and perhaps a cure for their particular condition. But while many studies are underway, to date, few drugs have…
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