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

182 evidence topics · 81 sources

Current treatment options and standard of care103 evidence topics · 55 sources
Place in treatment, anticipated use, and care setting17 evidence topics · 16 sources
Heterogeneity of treatment effect14 evidence topics · 9 sources
Care management intervention strategies21 evidence topics · 18 sources
Other product development or post-marketing obligations required by the FDA4 evidence topics · 5 sources
Ongoing post-approval monitoring6 evidence topics · 6 sources
Expected outcomes of therapy17 evidence topics · 14 sources