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VCP disease is a genetic disease that can affect the muscles, bones, nerves, and brain. Individuals with this condition typically develop symptoms in mid-adulthood and may only exhibit one symptom.
This condition is inherited in an autosomal dominant pattern, meaning an affected individual has a 50% chance of passing the VCP mutation to a child. Genetic testing is the best way to diagnose this condition.
Symptoms vary from person to person, even among family members. It is unknown how many people are affected by this condition, but it is extremely rare.
Patients can work with a team of doctors and therapists to develop an individualized, comprehensive care plan to screen and treat the various symptoms that may develop over a person’s lifetime. Currently, there is a treatment for Paget's disease of bone, and treatments for other symptoms are also in the pipeline. A care plan for a patient can include physical and speech therapy, prescriptions for mobility aids, monitoring for other diseases, and support for respiratory, pain, and mental health symptoms. These supportive therapies may improve quality of life.
This is no cure yet, but we’re working on it!
