DUX4

Chr 4

double homeobox 4

Also known as: DUX4L

This gene is located within a D4Z4 repeat array in the subtelomeric region of chromosome 4q. The D4Z4 repeat is polymorphic in length; a similar D4Z4 repeat array has been identified on chromosome 10. Each D4Z4 repeat unit has an open reading frame (named DUX4) that encodes two homeoboxes; the repeat-array and ORF is conserved in other mammals. The encoded protein has been reported to function as a transcriptional activator of paired-like homeodomain transcription factor 1 (PITX1; GeneID 5307). Contraction of the macrosatellite repeat causes autosomal dominant facioscapulohumeral muscular dystrophy (FSHD). Alternative splicing results in multiple transcript variants. [provided by RefSeq, Apr 2015]

GeneReviewsOMIMResearchGenerating clinical summary…

Primary Disease Associations & Inheritance

UniProtFacioscapulohumeral muscular dystrophy 1
7
Active trials
126
Pubs (1 yr)
P/LP submissions
P/LP missense
LOEUF
GOF
Mechanism· predicted
📖
GeneReview available — DUX4
Authoritative clinical overview · Recommended first read
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Population Genetics & Constraint

Constraint data not available from gnomAD.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

DUX4 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Facioscapulohumeral Muscular Dystrophy (FSHD)

A Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD

NOT YET RECRUITING
NCT07798609Phase PHASE1, PHASE2Soufflé Therapeutics, Inc.Started 2026-09
SFL-0821 for injectionPlacebo
Facioscapulohumeral Muscular Dystrophy Type 2

An 18-month Prospective Natural History Study to Gain Insight Into FSHD2 Pathophysiology and Disease Progression

RECRUITING
NCT06079567Phase NACentre Hospitalier Universitaire de NiceStarted 2023-10-03
Validation of new COMs for FSHD2 patients
Facioscapulohumeral Muscular Dystrophy

ADVANCED FSHD-COM: New Clinical Outcome Measures to Evaluate Non-ambulant FSHD Patients, a Pilot Study

ACTIVE NOT RECRUITING
NCT05453461Phase NACentre Hospitalier Universitaire de NiceStarted 2023-04-03
Validation of new COMs for non ambulant FSHD patients
Facioscapulohumeral Muscular Dystrophy 1

Study to Evaluate the Efficacy and Safety of Satralizumab in FSHD1

ACTIVE NOT RECRUITING
NCT06222827Phase PHASE2Centre Hospitalier Universitaire de NiceStarted 2024-01-24
Satralizumab Prefilled SyringePlacebo Comparator
Muscular Dystrophy, Facioscapulohumeral

Clenbuterol to Target DUX4 in FSHD

RECRUITING
NCT06721299Phase PHASE1Jeffrey StatlandStarted 2025-06-25
Clenbuterol
Muscular DystrophyFacioscapulohumeral

Clinical Trial Readiness Network FSHD France: Prospective 24 Months MRI Study

ACTIVE NOT RECRUITING
NCT04038138Phase NACentre Hospitalier Universitaire de NiceStarted 2019-09-16
Validation of new COA for FSHD patients
FSHD

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

RECRUITING
NCT04635891University of Kansas Medical CenterStarted 2020-12-15