Facioscapulohumeral muscular dystrophy (FSHD) is an inherited progressive muscle-wasting disease affecting the face, arms and shoulders. No targeted treatments are available for this debilitating condition, which is most commonly diagnosed in teenagers and young adults.
The FSHD Global Research Foundation grant is supporting an Institute team’s search for drugs that could halt the progression of FSHD.
The gene SMCHD1 is known to be faulty in people with a type of FSHD.
A research team including Professor Marnie Blewitt, Associate Professor James Murphy, Associate Professor Chris Burns and Dr Kelan Chen is working to design medicines that boost SMCHD1’s activity to correct the defect that causes FSHD.
With this grant, the team will screen a library of 120,000 drug-like molecules. Promising molecules can then be refined to develop potential drugs that might treat FSHD.
Ms Natalie Livet, CEO of FSHD Global Research Foundation, said her organisation was committed to advancing medical research and education into FSHD to find treatments, and ultimately a cure.
“We are thrilled to be funding this promising area of research at WEHI. Our hope is that it will lead to new therapies that could either halt the progression of FSHD, or potentially improve symptoms or even prevent the disease in people at genetic risk.”