FDA places clinical hold of RP-A501 for the treatment of patients with Danon disease due to a patient death. Click here to ...

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 ...

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.

The FDA ordered a clinical hold on Rocket Pharmaceuticals' Danon disease trial after the adverse event was first disclosed.

Rocket Pharmaceuticals halted its Danon disease gene therapy trial after a patient death, with the FDA placing the study on ...

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 ...

They report that in these cells LAMP2 fluorescence is higher but BMP fluorescence is lower, MVE size is reduced, and that MVEs contain less ILVs. They also report that LAMP2-positive EVs are increased ...

The collaborative research utilized a novel molecular dynamics simulation to examine the interaction between the parasite's protein gp82 and the human receptor LAMP2. This interaction is pivotal ...

The cause of Danon disease is a mutation in the LAMP2 gene on the X chromosome. Because it is an X-linked disorder, males are more severely affected than females since they only have a single X ...

We also found that 6-OHDA appeared to promote the unfolding of PARK7, allowing its selective recognition by the chaperone HSPA8 via KFERQ-like motifs, leading to PARK7 translocation to the lysosomal ...

Danon disease is an X-linked disorder that leads to fatal cardiomyopathy caused by a deficiency in lysosome-associated membrane protein-2 (LAMP2). In female patients, a later onset and less severe ...