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ACD-related telomere biology disorder

MONDO:0800469

A complex set of inherited conditions caused by a pathogenic variant(s) in the ACD gene that results in aberrant telomere biology.

Also known as: ACD-related telomeropathy

2 clinical trials for this condition and its sub-types, 0 tagged with ACD-related telomere biology disorder itself.

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Where it sits in the disease tree

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Part of

↑ Hereditary disease (18235) ↑ Telomere syndrome (92)

Sub-types of ACD-related telomere biology disorder

ACD-related long telomere syndrome 0 trials
ACD-related short telomere syndrome 0 trials
2 sub-types
- Dyskeratosis congenita, autosomal dominant 6 0 trials
- Dyskeratosis congenita, autosomal recessive 7 0 trials

Including sub-types (2) Tagged with ACD-related telomere biology disorder (0)

Not yet finished but already full! 1 Completed 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 1 • Sponsor: Elixirgen Therapeutics, Inc. • Aim: Disease control

Last updated Jun 27, 2026 09:02 UTC