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Cerebroretinal microangiopathy with calcifications and cysts 3

MONDO:0957264

15 clinical trials for this condition and its sub-types.

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Broader categories

Disease (680) Nervous system disorder (231) Hereditary disease (176) Eye disorder (102) Retinal disorder (85) Human disease (14) Telomere syndrome (9) Hereditary neurological disease (6) Premature aging syndrome (6) Disorder of orbital region (3)

Trials to join now! 6 Not yet recruiting 1 Not yet finished but already full! 1 Completed 6 Terminated 1

Experimental infusion aims to fix bone marrow failure in rare telomere disease

Disease control Ongoing

This early-stage trial tests a single infusion of EXG34217 in 12 people aged 12 and older who have bone marrow failure due to telomere biology disorders. The main goal is to check safety and tolerability, while also looking at changes in telomere length and blood cell counts. Bec…

Phase: PHASE1 • Sponsor: Elixirgen Therapeutics, Inc. • Aim: Disease control

Last updated Jun 27, 2026 09:02 UTC