Facioscapulohumeral muscular dystrophy causes muscle weakness and atrophy — starting with weakness of the face, shoulders and upper arms and eventually affects nearly all skeletal muscles.
It is a disorder that scientists currently have no cure for but associate professor of biochemistry and molecular biology at Saint Louis University, Fran Sverdrup, wants to change that.
The findings inspire hope to many who suffer from the cruel disease, which includes Fran’s daughter.
The findings were published in the Journal of Pharmacology and Experimental Therapeutics.
Fran along with his team have successfully stopped the expression of the protein in cell as well as animal models, according to the paper.
Fran has a daughter who was diagnosed with this uncurable illness, so he decided to find one.
“We have been working to find a safe drug that would ‘turn off’ the toxic gene that causes FSHD,” he revealed. “Now we have demonstrated that we can do this in an animal model with a drug that can be taken in pill form.”
Scientists have been searching for an avenue to prevent the gene’s expression — which would stop or revert the course of the disease.
Recently, Fran’s team discovered that this can be done by re-purposing an already existing drug.
He is encouraged as these new findings suggest a way in developing the first treatment or possible cure for FSHD.