They also hope to unlock the ability to pursue cures for polygenic diseases as well. Currently, Rocket is working on gene therapies for LAD-I, FA, PKD, Danon, PKP2-ACM, and BAG3-DCM.

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.

Additionally, Rocket has secured an ICD-10 code from CMS for LAMP2 deficiency in Danon Disease About RP-A501 RP-A501 is Rocket’s investigational gene therapy product for the treatment of Danon ...

Rocket Pharmaceuticals ... that the company will be presenting new data on the results of the RP-A501 Phase 1 trial to treat patients with Danon disease at the American Heart Association ...

COO and President of Rocket Pharmaceuticals, Kinnari Patel’s, shares how the company is using gene therapy to eradicate diseases in children. Newsletters Amazon Prime Day Share a News Tip.

Feb. 28 is Rare Disease Day. It's meant to observe the more than 400 million people globally — 50% of them children — that are affected by rare, pediatric life-threatening diseases such as ...

Chimpanzees share almost all their gene sequences with humans, and this closeness has made them ideal animal models for many human diseases. But similarities between humans and nonhuman primates go ...