Company-reported prevalence
RP-A501
Overview
| Field | Value |
|---|---|
| Drug | RP-A501 (AAV9.LAMP2B) |
| Indication | Danon disease |
| Disease | Danon disease (LAMP2 deficiency) |
| Company | Rocket Pharmaceuticals, Inc. (worldwide development and commercialization rights); REGENXBIO Inc. (licensor of the AAV9 vector under the 2018 exclusive worldwide license for Danon disease; no product rights) |
| Documents searched | Website pages: home, our disease focus, Danon disease clinical trial, Danon disease natural history study, Danon disease resources, genetic testing (Mission: Genome), healthcare professionals, clinical trials, and our science (accessed October 2026); investor website press release list and events and presentations page (accessed October 2026); press releases, September 17, 2024, September 15, 2026, and October 7, 2026, and financial results releases for the fourth quarter of 2025 and the first and second quarters of 2026; Form 10-K for 2025 (February 2026), for 2024 (February 2025), and for 2022 (February 2023); Danon disease program update presentation, October 2023; investor presentation, November 2024 (slides carry no text layer); corporate presentation, August 2026; Danon disease program update presentation, October 2026; Invitae Danon disease sponsored testing program page (accessed October 2026) |
| Data as of | October 14, 2026 |
| Prepared by | AI research agent (Claude); not yet reviewed by a person |
Company statements of prevalence
United States
Number affected: Rocket Pharmaceuticals Danon disease program update presentation, October 2026
Number affected: Rocket Pharmaceuticals press release, October 2026
Cited source: None stated in the document.
(opens the full-size image in a new tab)Number diagnosed: Rocket Pharmaceuticals Danon disease program update presentation, October 2026
Number affected: Rocket Pharmaceuticals corporate presentation, August 2026
“Est. US DD Population ~12,000 patients” (opens the source at this quote in a new tab)
“HCM with DD Population ~8,200 patients” (opens the source at this quote in a new tab)
Cited source: 2.6 Maron 2018; 2.7 Massera 2023; 2.8 Taylor 2025
(opens the full-size image in a new tab)Europe
No evidence found.
Global
No evidence found.
Other countries and regions
Number affected (United States and EU5): Rocket Pharmaceuticals Danon disease program update presentation, October 2026
Cited source: None stated in the document.
(opens the full-size image in a new tab)Prevalence: Rocket Pharmaceuticals Form 10-K for 2025, February 2026
Cited source: None stated in the document.
(opens the full-size image in a new tab)Prevalence: Rocket Pharmaceuticals corporate presentation, August 2026
Cited source: 2.9 Rocket Pharmaceuticals 2026b
(opens the full-size image in a new tab)Incidence: Rocket Pharmaceuticals corporate presentation, August 2026
Cited source: None stated in the document.
(opens the full-size image in a new tab)Geography not stated
No evidence found.
Sources the company cites
Genome aggregation database 2024
Reference as the company lists it
Passage carrying the figure
Not quoted: A population genomic database that a person must query; the presentation names no query, variant list, or address, and gives the population allele frequencies it derived without stating them.
Invitae, ambry genetics, and GeneDx 2026
Reference as the company lists it
Passage carrying the figure
Not quoted: Proprietary laboratory and natural history study records; no public document exists.
Cellworks and forian 2026
Passage carrying the figure
Not quoted: Commercial claims and electronic health record databases that a person must license and query; no public document exists.
Commercial genetic testing laboratories 2026
Passage carrying the figure
Not quoted: Purchased de-identified test data; the presentation names no laboratory for this footnote and no public document exists.
Rocket pharmaceuticals 2026a
Passage carrying the figure
Not quoted: Internal company modeling; no public document exists.
Maron 2018
Reference as the company lists it
Passage carrying the figure
Not quoted: The citation names no findable document; PubMed holds no article at Circulation 2018, volume 138, page 1522, and no 2018 Circulation article by Maron MS carries those pages.
Massera 2023
Reference as the company lists it
Passage carrying the figure
Taylor 2025
Reference as the company lists it
Passage carrying the figure
Not quoted: The poster abstract was not located in a public abstract archive.
Rocket pharmaceuticals 2026b
Reference as the company lists it
Passage carrying the figure
Not quoted: Company data on file; no public document exists.
References
- Massera 2023 (opens in a new tab)
- Rocket Pharmaceuticals (2026) corporate presentation, August 2026 (opens in a new tab)
- Rocket Pharmaceuticals (2026) Danon disease program update presentation, October 2026 (opens in a new tab)
- Rocket Pharmaceuticals (2026) Form 10-K for 2025 (opens in a new tab)
- Rocket Pharmaceuticals (2026) press release, October 7, 2026 (opens in a new tab)