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Ultragenyx Gene Therapy Becomes First FDA-Approved Treatment for Ultra-Rare DiseaseNews DeskAugust 20, 20260 An ultra-rare disease whose standard of care for decades has been consuming a kitchen pantry staple every few hours now…
PTC Therapeutics’ $211M Bid Wins Bankruptcy Auction for Sangamo Gene TherapyNews DeskAugust 13, 20260 PTC Therapeutics is expanding its prospects in rare disease, winning a bankruptcy auction for a Sangamo Therapeutics gene therapy that’s…