Healio9d
Q&A: ‘An exciting breakthrough’: Gene transfer therapy promising in Danon disease
Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...
2 Minute Medicine8d
AAV9.LAMP2B gene therapy shows clinical benefit in Danon disease
In seven patients with Danon disease, a single infusion of adeno-associated virus serotype 9 carrying transgene LAMP2B ...
Yahoo Finance23d
Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results and Highlights Recent Progress
Dosing ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease; Program update expected in the first half of 2025 New England Journal of Medicine publication of Phase 1 study of RP-A501 ...
TipRanks on MSN7d
Sunrun, HubSpot, Rocket, Summit, Cybin: Trending by Analysts
Analysts are intrested in these 5 stocks: ( ($RUN) ), ( ($HUBS) ), ( ($RCKT) ), ( ($SMMT) ) and ( ($CYBN) ). Here is a ...
pharmaphorum6d
Rocket Pharmaceuticals in orbit after gene therapy read-out
Results came from a phase 1 trial of RP-A501 for treatment of Danon Disease and sent shares up 75% on the Nasdaq to more than $56, a five-year high. The surging stock price indicates the market's ...