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Families step up to find gene therapies for diseases too rare for research firms

When a disorder affects only a few people around the world, companies see little incentive to find out what could treat it. Increasingly, advocacy networks are funding and promoting their own research ....

United States , Christopher Espana , Ronald Cohn , Terry Pirovolakis , Stephanie Telesca , Precision Child Health , Canadian Organization For Rare Disorders , Log Increate Free , Mount Sinai Hospital , Canadian Organization , Rare Disorders , Sick Children , Elpida Therapeutics ,