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MacTel2: Vascular disease or neurodegenerative disease?
0:39
MacTel2: Vascular disease or neurodegenerative disease?
2 days ago
by
Robin Vora, MD(+1 more)
Retina as a window to the brain: a biomarker approach to ROP
0:57
Retina as a window to the brain: a biomarker approach to ROP
6 days ago
by
Cytnthia Toth, MD(+2 more)
QUASAR 64-week results: aflibercept 8 mg in macular edema following RVO—Jordana G. Fein, MD, MS
1:21
QUASAR 64-week results: aflibercept 8 mg in macular edema following RVO—Jordana G. Fein, MD, MS
16 days ago
by
Jordana Fein, MD, MS(+1 more)
Can a retinal scan predict autism risk?
0:42
Can a retinal scan predict autism risk?
20 days ago
by
Cytnthia Toth, MD(+2 more)
How OLN324 differs from faricimab
0:51
How OLN324 differs from faricimab
22 days ago
by
Veeral Sheth, MD, MBA, FACS, FASRS(+1 more)
Arshad Khanani, MD, MA, FASRS, discussed the real-world implications of SOL-1 data for AXPAXLI in wet AMD
0:41
AXPAXLI's real-world potential to reduce wet AMD treatment gaps
a month ago
by
Arshad M. Khanani, MD, MA, FASRS(+2 more)
Sandeep Dev, MD, FASRS, on AI's future role in retina care
0:24
AI's future role in retina care recordkeeping, imaging, and research
a month ago
by
Sundeep Dev, MD, FASRS(+2 more)
Treatment durability and Medicare status both drive retinal outcomes
0:38
Treatment durability and Medicare status both drive retinal outcomes
a month ago
by
Sundeep Dev, MD, FASRS(+1 more)
Sundeep Dev, MD, FASRS on faricimab timing and durability
0:24
Early faricimab use linked to longer treatment durability
a month ago
by
Sundeep Dev, MD, FASRS(+2 more)
Patient education in the AI era
0:47
Patient education in the AI era
a month ago
by
Margaret Chang, MD, MS(+2 more)

CME Content


Danon disease is a rare, multisystem X-linked genetic disorder caused by variants in the LAMP2 gene. In children and young adults, it is defined overwhelmingly by its cardiac effects . What is far less appreciated is that the eye is frequently involved and that the retinal findings can appear before the systemic diagnosis is established.

Rare retinal diseases initially were the primary targets of gene therapy, but that is no longer the case. The focus has expanded beyond those to also include more common, chronic diseases such as diabetic macular edema and wet age-related macular degeneration. During the first half of 2026, that trend was evident.