Conquering Diseases

Study Of An Investigational Gene Therapy Drug For Boys With Duchenne Muscular Dystrophy (Dmd)

Description

Seeking boys between the ages of four and 17 with Duchenne Muscular Dystrophy (DMD) for a study of an investigational gene therapy drug that may improve their body's ability to make dystrophin, a protein that helps protect muscles from stress and damage.

Overview

We are studying how safe and whether the investigational therapy has the potential to treat certain boys with DMD. The study has two parts, a screening period and a dosing and follow-up period. Each volunteer who completes the screening will receive the investigational drug at either a high or low dose. Boys cannot be on certain medications or supplements while on the study.

What we're hoping for

We hope that the investigational drug will be safe to use and slow muscle damage and reduce the overall loss of muscle function in boys with DMD

Additional Information

ClinicalTrials.gov Identifier: NCT03368742

 Principal Investigator

Brenda  Wong, MD

UMass Chan Medical School

 Study Contact

Sarah  Figueira

508-856-1604

sarah.figueira@umassmed.edu

 Location

UMass Chan University Campus

55 Lake Ave North 

Worcester,MA 01655

508-856-8989