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.
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
ClinicalTrials.gov Identifier: NCT04003974