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0.000706380974805718 0.000205078347524175 -0.00423069035448171 -0.00650933866030678 -0.00233181676629433 -0.00461046507211927 -0.000432943178106817 -0.000432943178106817
Stock impact report

Rocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments: Analyst [Yahoo! Finance]

Rocket Pharmaceuticals, Inc. (RCKT) 
Last rocket pharmaceuticals, inc. earnings: 3/5 07:00 am Check Earnings Report
US:NASDAQ Investor Relations: rocketpharma.com
Company Research Source: Yahoo! Finance
Danon disease is a rare X-linked dominant genetic disorder that manifests with the clinical triad of cardiomyopathy (stiff heart muscles), skeletal myopathy (weakness), and intellectual disability. Also Read: FDA Rejects Rocket Pharmaceuticals' Gene Therapy For Rare Immune Disorder, Asks For More Data, Stock Slides Danon disease is caused by mutations in the LAMP2 gene, which contains instructions for the production of an enzyme called lysosomal-associated membrane protein-2 (LAMP-2). The data showed that RP-A501 was generally well tolerated. All evaluable Danon disease patients demonstrated LAMP2 protein expression at 12 months (sustained up to 60 months) and reduction of left ventricular (LV) mass index by =10% at 12 months (sustained up to 54 months) after treatment. These data were presented at the American Heart Association (AHA) Scientific Sessions 2024. Evidence of sustained clinically meaningful improvement was observed in pediatric patients followed up to 24 months Show less Read more
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