Rocket Pharmaceuticals is also progressing in its other projects, like the rolling BLA for Fanconi anemia and the Phase 1 enrollment completion in PKP2-arrhythmogenic cardiomyopathy. Furthermore, the ...
Rocket Pharmaceuticals, Inc. , a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that ...
Background: The exact mechanism of hyperammonemia is thought to be multifactorial, but is not yet fully understood. No studies have yet reported hyperammonemia combined with Fanconi syndrome caused by ...