Minneapolis, MN - Mutations in the lysosome-associated membrane protein (LAMP2) gene result in a cardiomyopathy characterized by clinical deterioration and early death in young patients, a new report ...
Rocket Pharmaceuticals (RCKT) presented long-term safety and efficacy results from the Phase 1 RP-A501 study which showed that RP-A501 was generally well tolerated and all evaluable Danon disease ...
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