welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- login
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.
other Duchenne content
-
Therapeutic Potential of Stem Cell in the Treatment of Duchenne Muscular DystrophyHere, we will describe the therapeutic p...
-
Why my patient advocacy organization is investing in CRISPRIn last year’s action film “Rampage,...
-
Santhera Enters into Agreement to Acquire Option from Idorsia for Exclusive Sub-License of First-in-class Dissociati...Santhera Pharmaceuticals and Idorsia Ltd...
-
Personalized gene and cell therapy for Duchenne Muscular DystrophyDystrophinopathies are diseases caused b...
-
Clinical trial shows some promise for Duchenne muscular dystrophy drugThe results of an international clinical...
-
Impact of three decades of improvements in standards of care on clinical outcomes in Duchenne muscular dystrophyObjective: The last three decades have ...
-
Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular DystrophyThe study is to demonstrate non-inferior...
