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ACD-related short telomere syndrome

MONDO:0100569

A spectrum of conditions, including dyskeratosis congenita, Hoyeraal-Hreidarsson syndrome, hereditary aplastic anemia, and pulmonary fibrosis, typically characterized by shortened telomeres due to a pathogenic variant(s) in ACD that results in impaired telomere maintenance.

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

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Sub-types

Dyskeratosis congenita, autosomal dominant 6 (0) Dyskeratosis congenita, autosomal recessive 7 (0)

Broader categories

Disease (618) Hereditary disease (172) Human disease (14) Telomere syndrome (9) Premature aging syndrome (6) Disease of genetic or genomic mechanism (2) ACD-related telomere biology disorder (0) Disease by developmental or physiological process (0) Disease by etiologic mechanism (0)
Trials to join now! 1 Not yet finished but already full! 1 Completed 1
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  • 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

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