Gene therapy clinical trials
Gene: DMD

DMD (dystrophin)
EnsemblGeneIds (GRCh38): ENSG00000198947
EnsemblGeneIds (GRCh37): ENSG00000198947
OMIM: 300377, Gene2Phenotype
DMD is in 19 panels

1 review

Ellen McDonagh (Genomics England Curator)

Green List (high evidence)

Drug: AVI-4658 (PMO) (intramuscular administered morpholino oligomer directed against exon 51) - trials https://ClinicalTrials.gov/show/NCT00159250 and https://clinicaltrials.gov/ct2/show/NCT00844597, Biological: BMS-986089|Biological: Placebo for BMS-986089 - https://ClinicalTrials.gov/show/NCT03039686
Created: 12 May 2018, 8:57 a.m.

Phenotypes
Duchenne Muscular Dystrophy

Created: 12 May 2018, 8:57 a.m.

Panel version: 0.6

Details

Sources

Expert Review Green
ClinicalTrials.gov

Phenotypes

Duchenne Muscular Dystrophy

Tags

Skewed X-inactivation

OMIM

300377

Clinvar variants

Variants in DMD

Penetrance

None

Panels with this gene

History Filter Activity

2 Jun 2020, Gel status: 3

Added Tag

Sarah Leigh (Genomics England Curator)

Tag Skewed X-inactivation tag was added to gene: DMD.

12 May 2018, Gel status: 4

Added New Source

Ellen McDonagh (Genomics England Curator)

DMD was added to Gene therapy clinical trials panel. Sources: ClinicalTrials.gov,Expert Review Green