AAV9.LAMP2B gene therapy for Danon disease: from early clinical experience to evolving safety and trial design considerations
Alessia Argirò , Iacopo Olivotto
Rare Disease and Orphan Drugs Journal ›› 2026, Vol. 5 ›› Issue (3) : 30
| [1] |
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| [2] |
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| [3] |
|
| [4] |
|
| [5] |
|
| [6] |
|
| [7] |
|
| [8] |
|
| [9] |
|
| [10] |
|
| [11] |
|
| [12] |
Rocket pharmaceuticals provides update on phase 2 clinical trial of RP-A501 for Danon disease. Rocket Pharmaceuticals, Inc. Available from: https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-provides-update-phase-2-clinical-trial-rp/ [Last accessed on 22 Sep 2026]. |
| [13] |
|
| [14] |
Rocket pharmaceuticals announces positive clinical safety update from initial three patients treated with RP-A501 Under modified phase 2 protocol for Danon disease. Available from: https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-positive-clinical-safety-update [Last accessed on 22 Sep 2026]. |
| [15] |
|
| [16] |
Rocket pharmaceuticals announces FDA has lifted the clinical hold on the pivotal phase 2 trial of RP-A501 for the treatment of danon disease. Rocket Pharmaceuticals, Inc. Available from: https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-fda-has-lifted-clinical-hold/ [Last accessed on 22 Sep 2026]. |
| [17] |
Rocket pharmaceuticals reports second quarter 2026 financial results and highlights recent progress. Rocket Pharmaceuticals, Inc. Available from: https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-reports-second-quarter-2026-financial/ [Last accessed on 22 Sep 2026]. |
| [18] |
|
| [19] |
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| [20] |
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