orphan drugs

Substrate placeholder — needs review · Wikimedia Commons (CC BY-SA 3.0)

Recent Advances in Gene Therapy for Rare Diseases Reported

Developments in gene therapy and targeted medications have shown progress in treating rare diseases such as spinal muscular atrophy and certain forms of blindness. These conditions affect an estimated 30 million people in the United States, with over 7,000 identified rare disease…

1 source