An ultra-rare disease affects fewer than 1 in 50,000 people and is among the toughest challenges in medicine, with very limited treatment options. In the U.S., fewer than 7,000 people live with any given ultra-rare condition.
The company we are highlighting today, X4 Pharmaceuticals Inc. (XFOR), developed the first and only FDA-approved therapy for one such ultra-rare disease and is now exploring the drug's potential in additional indications.
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