Muscular dystrophy is rare progressive neuromuscular disease which affects nerves, tissue and muscles causing them to waste away. Often muscular dystrophy is diagnosed at childhood, some types emerge in adulthood. Due to the nature of the disease, patients experience muscle wastage and loss which can severely affect their movement ability and sometimes their ability to breathe. Whilst there is no current cure for muscular dystrophy, there are a number of treatments available which can improve a patient’s quality of life. Clinical trial research continues to explore new treatments and procedures to help support patients and caregivers. There are over 30 different sub disease categories of muscular dystrophy. Some of the most common include:
Duchenne Muscular Dystrophy (DMD) is a hereditary condition, caused by different mutations changes in DNA within the dystrophin gene which affects the muscles, causing them to become weaker and deteriorate over time. In Europe and North America, Duchenne Muscular Dystrophy affects 6 out of every 100,000 individuals.
Becker Muscular Dystrophy (BMD) is a genetic condition characterized by progressive muscle weakness. Whilst similar to Duchenne muscular dystrophy (DMD), it can have a later onset and milder symptoms compared to DMD. It is estimated that Becker muscular dystrophy occurs in approximately 1 in 30,000 male births.
Limb-girdle muscular dystrophy (LGMD) refers to a group of diseases that cause weakness and wasting of the muscles, specifically muscles of the shoulders, upper arms, pelvic area, and thighs. Estimates suggest that it affects around 0.8 to 6 people per 100,000 individuals worldwide.
Myotonic dystrophy (DM) is a rare genetic multi-system disorder. This means its symptoms can affect, as well as muscle wastage, various organs and tissues such as the heart, lungs, the digestive system, eyes, hormone-producing glands and the brain. It is estimated to affect 1 in 2,100 people or over 3.6million individuals worldwide.
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18 Jan 2024
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MDA is a health nonprofit advancing research, care and advocacy for people living with muscular dystrophy, ALS, and related neuromuscular diseases.
PPMD accelerates research, raises voices to impact policy, demand optimal care for every single family, and strives to ensure access to approved therapies.
The Akari Foundation educate and empower the Hispanic community about rare diseases, help with resources, awareness, advocacy, and education, entirely in Spanish, specializing in Duchenne muscular dystrophy.
A clinical trial is a research study that examines on volunteers the effects of a medical treatment, device, or other intervention, which is not yet approved. Its purpose is to assess, prior to its potential approval, the safety and effectiveness of the treatment for a specific health condition before it becomes widely available. Besides focusing on treatment, clinical trials also explore new approaches to diagnosing and preventing diseases.
Individuals with specific health conditions, such as muscular dystrophy, may qualify to participate in clinical trials. Researchers establish criteria for participation, which can be based on factors like age or the presence of other medical conditions. To determine eligibility, participants may need to answer questions or undergo a medical examination. Some trials also include healthy individuals without any known health conditions, while others may involve people with a family history of a particular disease or condition.
We make it straightforward for patients and their physicians to discover and receive support with accessing clinical trials. We empower patients and their families to know and understand their options and make the best possible decisions regarding their treatment journey.
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