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I have voluntarily chosen to provide this personal information, including contact information. By providing this information, I am authorizing PTC Therapeutics, Inc. and its agents and contractors (“PTC Therapeutics”) to contact me about treatments, products, and services related to the treatment of Duchenne muscular dystrophy (DMD), as well as to use and disclose the information for other purposes described below, using the contact information I have provided. I understand and agree that PTC Therapeutics may leave messages for me that disclose that someone has DMD or takes specific medication or that a specific medication used to treat DMD is available.

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Date of preparation: September 2022
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Important Terms and Concepts

Basic terms and key definitions

Neuromuscular disorders

Neuromuscular disorders affect the muscles and nerves, and most of the causes are genetic. This means they are either passed down through the family or caused by changes in an individual person’s genes.

Most neuromuscular disorders cause muscle weakness that worsens over time. Signs and symptoms of neuromuscular diseases can vary and may be mild, moderate, or severe.

Most often, when a child has a neuromuscular disease, they don’t grow and develop as fast as other children their age. They are often slow to start lifting their head, sitting, walking, and talking.

Treatment and supportive care may improve the symptoms of a neuromuscular disorder, increasing mobility and even life expectancy.

Muscular dystrophy

Muscular dystrophy is the term for a group of neuromuscular disorders that cause muscle weakness and muscle loss.

Duchenne muscular dystrophy (DMD) is a type of muscular dystrophy that causes muscle weakness that worsens over time. The progression and symptoms can vary from person to person.