Glycogen Storage Disease Type IIb – Talk Bio

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Rocket Pharmaceuticals Cuts 30% of Staff, Refocuses on Heart Gene Therapies

17 hours ago talkbio0Tagged 30%, Anemia, BAG3-associated, cardiovascular gene therapy, Deficiency of pyruvate kinase, Dependovirus, Financial cost, Glycogen Storage Disease Type IIb, Kresladi, layoffs, PKP2-associated cardiomyopathy, restructuring, Rocket

Rocket Pharmaceuticals; layoffs; restructuring; 30% workforce reduction; cardiovascular gene therapy; adeno-associated virus (AAV); Danon disease; PKP2-associated cardiomyopathy; BAG3-associated dilated cardiomyopathy; operating cost reduction; Fanconi Anemia; Pyruvate Kinase Deficiency; KRESLADI

Patient Death in Rocket Pharmaceuticals Gene Therapy Trial Prompts FDA Clinical Hold

2 months ago talkbio0Tagged acute systemic infection, Adverse event, Capillary Leak Syndrome, gene therapy, Glycogen Storage Disease Type IIb, Patient Death, Rocket, RP-A501, Study on Hold, United States Food and Drug Administration

Rocket Pharmaceuticals; gene therapy; Danon disease; clinical hold; FDA; patient death; capillary leak syndrome; acute systemic infection; RP-A501; adverse event