In seven patients with Danon disease, a single infusion of adeno-associated virus serotype 9 carrying transgene LAMP2B ...

Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...

We came across a bullish thesis on Rocket Pharmaceuticals, Inc. (RCKT) on Twitter by sharkbiotech. In this article, we will ...

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today ...

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 ...

RP-L102 for Fanconi Anemia and RP-A501 for Danon Disease show strong clinical data, indicating significant market potential. Financially, Rocket Pharma has a limited cash runway of 2–4 quarters ...

In 2024, we made strong progress in advancing our gene therapy pipeline, underscored by the New England Journal of Medicine publication of the ...

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 ...