Rocket Pharmaceuticals has received the green light from the US Food and Drug Administration (FDA) to resume its pivotal Phase II Danon disease gene therapy trial, according to a recent…
During clinical trials, the FDA may sometimes order a clinical hold. This order is often given to halt or suspend trial operations if the FDA believes that participants would…
Rocket Pharmaceuticals has recently dosed the first patient in their trial of RP-A501, a treatment for Danon disease. According to BioSpace, this trial will test the safety, tolerability, and efficacy…
Danon Disease Danon disease (DD) is an extremely rare condition that is not very well understood in the medical community. It was first described in 1981, and it is named…
Danon disease is a rare condition caused by a mutated LAMP-2 gene. This gene has three different forms- LAMP-2A, 2B, and 2C. Depending in which variant is mutated, individuals with…
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