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Glycogen Storage Disease

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Finding out your child has a rare genetic condition that few healthcare providers treat can be mind-boggling.

Glycogen storage disease (GSD) is one of those rare conditions. If your child has it, your emotions are probably all over the place. And you likely have a million questions about treatment and your child’s future. Because GSD is so rare, you’ll want to find a provider who knows what it takes to treat it.

You don’t have to look far. Cleveland Clinic Children’s has one of the most comprehensive programs for diagnosing and treating children with GSD. With early diagnosis and treatment, your child can live an active, healthy life.

Why Choose Cleveland Clinic Children’s for Glycogen Storage Disease Care?

Trusted experts:

Our experienced gastroenterologists (digestive system experts) treat many babies and children with glycogen storage disease. We can teach you what you need to know about GSD and offer your child the latest treatments and therapies.

Specialized knowledge:

Our providers stay current with the latest gene therapies and can let you know if your child can take part in a clinical trial or other new treatment options.

Personalized care:

There are 15 types of glycogen storage disease. Our team helps you understand what’s causing your child’s GSD. Our expert team of providers and dietitians work together to create a custom treatment plan based on your child’s diagnosis. Meet our team.

Recognized expertise:

Cleveland Clinic Children's has one of the largest glycogen storage disease programs in the nation. People come from all over the country to meet with our specialists who have in-depth knowledge of metabolism (how your body changes the food you eat into energy) disorders related to the digestive system.

National recognition:

Cleveland Clinic Children’s is a trusted healthcare leader. We’re recognized throughout the U.S. for our expertise and care.

Virtual visits:

We want to make getting care for your child as easy and convenient as possible. That’s why we offer virtual visits for follow-up care and if you live out of state. All you need is a smartphone, computer or tablet, and you can meet with your child’s provider online, from the comfort of home.

Glycogen Storage Disease Diagnosis at Cleveland Clinic Children’s

There are many different types of glycogen storage disease. Most affect the liver and muscles, but some can affect other parts of your child’s body, like their kidneys, heart, blood cells and nervous system.

Our providers will usually do an ultrasound and a variety of blood tests to better understand what’s happening in your child’s body.

Our program coordinators will help you schedule your first visit, where we’ll go over your child’s lab test results. There’s a good chance we’ll have your child stay overnight so we can keep an eye on your child’s glucose (sugar), LDH (lactate dehydrogenase) and ketone (what your liver makes when it breaks down fats) levels.

Providers Who Treat Glycogen Storage Disease

Locations

Our healthcare providers see patients at convenient locations throughout Northeast Ohio.

Glycogen Storage Disease Treatment at Cleveland Clinic Children’s

GSD is a chronic condition, but our team can help your child learn to manage it. We’ll create a personalized treatment plan just for them. They’ll most likely need to test their blood glucose levels throughout the day to avoid hypoglycemia (low blood sugar).

Depending on what type of GSD they have, treatment can include:

Special GSD diet

Your child may be able to keep a steady glucose level by eating uncooked cornstarch throughout the day. Because cornstarch is a complex, slow-release carbohydrate, it helps blood glucose levels stay stable.

Other options may include eating high protein foods and having small meals every two to four hours throughout the day. Your child might also need to limit sugars and take vitamin D, calcium and iron supplements.

Medications

Some medications can manage the side effects of glycogen storage disease by:

Reducing uric acid levels in the blood, which helps manage symptoms of arthritis that can develop in children or teens with GSD type I.
Lowering lipid levels.
Treating recurring infections that often happen in people with GSD type Ib.

Gene therapy

Besides medications, there are also promising new gene therapy treatments for glycogen storage disease. Our specialists keep up with all the latest clinical trials and treatments. And they’ll let you know if your child can take part in a clinical trial.

Organ transplant

In rare cases, some children may need a liver transplant and/or kidney transplant. This is often the last resort when GSD has badly damaged their liver or kidney. If your child needs a transplant, know that our specialists will be right by your side throughout the entire process.

Taking the Next Step

Coping with a rare genetic condition can be challenging, especially when it involves your child. But at Cleveland Clinic Children's, you won’t have to handle things alone. Our team of experts will surround your child (and your family) with expert medical care, compassion and support. We’ll find the right treatment plan that fits your child’s unique needs. And we’ll help your child manage the condition so they can live an active, healthy life now and in the future.