As Edgewise Therapeutics works towards its first FDA drug approval, the small biopharma is already launching resources for one of the rare disease communities it aims to help.
The disorder is Becker muscular dystrophy, which affects about 5,000 people in the US and often sits in the shadow of the better-known condition Duchenne muscular dystrophy. The conditions are similarly the result of protein mutations that cause progressive muscle damage, but Becker symptoms typically appear later in life. Duchenne’s patients often start showing symptoms by 1 to 2 years old.
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