Conquering Diseases

Study Of An Experimental Drug In Treating People With Facioscapulohumeral Muscular Dystrophy (Fshd)

Description

Facioscapulohumeral muscular dystrophy 1 (FSHD1, also known as Landouzy-Dejerine’s disease) is a genetic disorder that affects how muscles work. We are seeking volunteers for a study of an experimental drug that may prevent or decrease the protein that impacts muscles in people with FSHD.

Overview

We are studying whether the experimental drug prevents or decreases the protein that causes muscles to die in people with FSHD. Some volunteers will get the experimental drug and others will get a drug that contains no medicine (placebo) so we can compare to see if the experimental drug reduces the level of proteins.

What we're hoping for

We hope that the experimental drug can be taken safely and decreases or prevents the protein that causes muscles to die in people with FSHD

Additional Information

ClinicalTrials.gov Identifier: NCT04003974

 Principal Investigator

Lawrence  Hayward, MD, PhD

University of Massachusetts Medical School

 Study Contact

Catherine  Douthwright

508-856-6491

Catherine.Douthwright@umassmed.edu

 Location

UMMS University Campus

55 Lake Ave North 

Worcester,MA 01655

508-856-8989