Clinical Trial

Disease: Duchenne Muscular Dystrophy, DMD, (NCT05514249)

Disease info:

Duchenne muscular dystrophy (DMD) is a rare genetic disease that is characterised by severe and progressive skeletal and cardiac muscle weakness and atrophy in the male population and less severe muscle weakness in the female population.

The disease often debuts between the ages of three and six years. Because of the rapid progressive nature of the disease, most patients require assistance in the form of a wheelchair by their teenage years, as the disease gradually extends to more muscle groups.

In addition to the decay and necrosis of muscle fibers, children suffering from DMD are affected by reduced bone density making them prone to fractures, especially in the hips and spine.

Patients of DMD are also at risk of cardiompyopathy, a heart disease that weakens cardiac muscles preventing proper heartbeat and blood pumping.

Duchenne muscular dystrophy affects about 1 in 3,500 to 1 in 9,300 males worldwide. The symptomatic prevalence in the female population is unknown
Official title:
Treatment of a Single Patient With CRD-TMH-001

Principal Investigator: Brenda Wong, MD


Cure Rare Disease, Inc


University of Massachusetts, Worcester


United States, Massachusetts

UMass Chan Medical School, Worcester, Massachusetts, United States, 01655

Study start:
Aug. 31, 2022
1 participant
Gene editing method:
Type of edit:
Gene correction
Delivery method:
Recombinant adeno-associated viral vector (rAAV) - In-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active not recruiting


The objective of the study is to assess the safety and preliminary efficacy of CRD-TMH-001 after intravenous administration for a period of 1 year with long-term follow-up out to 15 years.

Last updated: Sep. 15, 2023
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