Duchenne | and you

Seeking professional help EARLY is critical

Duchenne can cause irreversible muscle damage, so early treatment is essential

Welcome to Duchenne and You

Duchenne and You is a resource dedicated the people who’s lives have been touched by Duchenne muscular dystrophy (DMD).

We are here to help guide you through the available information on DMD so you or your loved one can focus on living the best life possible.

We will help you understand what is happening and what to expect, to help you decide what to do next.

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This site is intended for US residents only.
The information on this site is not intended to make a diagnosis or to take the place of talking to a US health care professional.

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© 2022 PTC Therapeutics, Inc. All rights reserved.

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.