Jan. 30 at 8:12 PM
$RCKT they are well into their 6 year at this point
The longest an individual patient has remained event-free after receiving RP-A501 is 5 years (60 months).
As of the most recent data published in the New England Journal of Medicine in November 2024, the primary results from the Phase 1 trial show:
Long-Term Survival: The first patient treated in the study reached the five-year follow-up mark with preliminary evidence of robust, sustained Grade 3 LAMP2B protein expression in the heart.
Transplant-Free Status: All six evaluable patients from the Phase 1 study—now up to age 25—remained alive and transplant-free for a follow-up period ranging from 24 to 54 months.
Comparison to Natural History
This 5-year event-free period is significant because, in untreated male patients, Danon disease is typically aggressive and fatal, with a median survival age of only 19 years. The current Phase 1 cohort includes patients surviving well into their mid-20s without needing a transplant
BULLISH.
