Muscular Dystrophy

Muscular dystrophy (MD) is a general term for a group of genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. The most common is Duchenne, which primarily affects male children, progresses rapidly, and leads to an inability to walk by early adolescence and need for a respirator by age 20.

Commonly Prescribed (On-Label) Drugs: None

Off-Label Prescription Drugs Breakthrough Options: Albuterol, Prednisolone, Prednisone

Myotonic Dystrophy

Myotonic dystrophy (MMD) is one of a group of genetic diseases involving progressive weakness and degeneration of the skeletal muscles that control movement. People with MMD have a better prognosis than those with muscular dystrophy. However, they have prolonged muscle spasms in the fingers and facial muscles; a floppy-footed, high-stepping gait; cataracts; cardiac abnormalities; and endocrine disturbances.

Commonly Prescribed (On-Label) Drugs: None

Off-Label Prescription Drugs Breakthrough Options: Mexiletine, Nifedipine

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