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Small Molecule Therapies


Muscular dystrophy is the name given to a group of inherited diseases characterized by progressive muscle weakness and wasting. Current therapies can help manage the symptoms of the disease, and slow down its progression, but cannot cure it. However, there are several experimental treatments such as small molecule therapies that are being investigated and have the potential to vastly improve the symptoms.

Small molecules are compounds with low molecular weights that can easily enter cells and directly affect the target, which may be a protein or other molecules within the cell. More than 90% of the therapeutics made today are small molecules.

Some of the small molecules being studied as potential muscular dystrophy therapies are summarized below, grouped by their mechanism of action.

Small Molecule Therapies