Health Canada Approves AGAMREE® (vamorolone) as the First Treatment for Duchenne Muscular Dystrophy in Canada [Yahoo! Finance]
Catalyst Pharmaceuticals, Inc. (CPRX)
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Source: Yahoo! Finance
treatment of Duchenne Muscular Dystrophy (DMD) in boys age 4 and older. This approval – granted under Health Canada's Priority Review process – marks an important milestone making AGAMREE ® the first treatment in Canada approved for DMD a muscle wasting disease that affects predominantly young boys. DMD is a rare, debilitating and life-shortening neuromuscular disease. The Canadian Neuromuscular Disease registry estimates that there are more than 800 boys and young men living with DMD in Canada and coping with its debilitating symptoms. DMD is a type of muscular dystrophy that causes weakness and a wasting of the muscle due to the absence of a protein called dystrophin. If the body is lacking dystrophin, muscle cells become damaged and movements that are considered natural such as getting up from the floor and walking become challenging. Boys with DMD typically lose the ability to walk in their early teens and have increasing cardiac and respiratory difficulties. Bone thinning and sco
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