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News & Videos

 UCSD's Dr. Adler Awarded $1.4M for stem cell research 





Young patient's skin cells used as a heart model





 Dr. Adler on the treatment of Danon Disease





FDA clears Rocket Pharma's IND for RP-A501





Rocket Pharmaceuticals Announces Preclinical Data for AAV-Based RP-A501, the First Investigational Gene Therapy Program for a Monogenic Heart Failure Syndrome





Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial of RP-A501 in Danon Disease




Gene Therapy for Male Patients With Danon Disease Using RP-A501; AAV9.LAMP2B