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An Evolving Regulatory Environment for Rare and Orphan Diseases

Advarra

The law established the Office of Orphan Products Development , providing financial incentives for pharmaceutical companies to develop orphan drugs and making it more viable to invest in orphan drug research and development. The impact was clear: Between 1983 and 2020, FDA approved 599 orphan drugs.

Disease 52
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Deliberate Dysentery

Codon

yellow fever, malaria, and dengue), evaluating new drugs or treatments, and studying pathogenesis, the process by which a disease develops. Scientists studied black men infected with syphilis for several decades while withholding known treatments. Now, we have upwards of 98 or 99 percent cure rates in some cases.

Vaccine 108