RP-A501 (AAV9.LAMP2B)

Danon disease

Manufacturer: Rocket Pharmaceuticals
Regulatory submission: BLA planned for late 2028
Launch: Anticipated in mid-2029
Last updated: October 8, 2026

Section

Product information and disease description Place of product in therapy Approaches to treatment

Current treatment options and standard of care

103 evidence topics · 55 sources