News

Mother and son inspire hope on World Transplant Day
Brittany Clayborne remembers the day her health crisis began in 2010. She had just given birth to her son, Micah, and was ...
Rocket Pharma Reports Patient Death In Rare Disease Gene Therapy Trial, FDA Puts Clinical Hold
Rocket Pharmaceuticals halted its Danon disease gene therapy trial after a patient death, with the FDA placing the study on ...
GEN - Genetic Engineering and Biotechnology News9d
Danon Disease Patient Dies in Rocket Gene Therapy Trial
Rocket Pharmaceuticals acknowledged the death of a patient in a pivotal trial assessing its Danon disease gene therapy candidate RP-A501, a study that the FDA has placed on clinical hold.
Rocket Pharmaceuticals: Despite FDA Clinical Hold On RP-A501, Prospects Remain Intact
FDA places clinical hold of RP-A501 for the treatment of patients with Danon disease due to a patient death. Click here to ...
Pharmabiz9d
US FDA places clinical hold on Rocket Pharma's gene therapy phase 2 trial of RP-A501 to treat Danon disease
US FDA places clinical hold on Rocket Pharma's gene therapy phase 2 trial of RP-A501 to treat Danon disease: Cranbury, New Jersey Wednesday, May 28, 2025, 14:00 Hrs [IST] Rocket P ...
Clinical Trials Arena on MSN10d
Patient dies in Rocket’s Phase II Danon disease gene therapy trial
The FDA ordered a clinical hold on Rocket Pharmaceuticals' Danon disease trial after the adverse event was first disclosed.
Le Lézard10d
Rocket Pharmaceuticals Provides Update on Phase 2 Clinical Trial of RP-A501 for Danon Disease
Rocket Pharmaceuticals, Inc. , a fully integrated, late-stage biotechnology company advancing an integrated pipeline of genetic therapies for rare diseases, today announced an update related to ...
Morningstar10d
Rocket Pharmaceuticals Provides Update on Phase 2 Clinical Trial of RP-A501 for Danon Disease
To support accelerated approval, the study assesses the efficacy of RP-A501 as measured by the biomarker-based co-primary endpoint consisting of improvements in LAMP2 protein expression ...