Glycogen Storage Disease III (GSDIII) Clinical Trial Opportunities

Patient with Glycogen Storage Disease Type III (GSDIII) Patient with Glycogen Storage Disease Type III (GSDIII)

Learn how you or a loved one might participate in research studies

About Glycogen Storage Disease III (GSDIII)

GSDIII is caused by a glycogen debranching enzyme deficiency that results in glycogen accumulation in the liver and muscles, disrupting the regulation of glucose. This can lead to extensive damage to the liver and muscles over time.

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Who is Ultragenyx?

At Ultragenyx, our goal is to bring novel therapies to patients with serious rare and ultra-rare genetic diseases. Since our founding in 2010, our team has had a proven track record of identifying, developing, and launching medicines for rare diseases. During this time, we’ve launched 3 FDA-approved treatments in 4 indications and developed a diverse portfolio of investigational therapies. We have deep expertise in multiple therapeutic areas, such as endocrine/bone diseases, metabolic diseases, and central nervous system (CNS)/muscle diseases. Most importantly, we put patients and their caregivers at the center of our work. All our efforts are rooted in our commitment to provide patients and their families with hope today.

Next steps

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Why are clinical trials important?

Clinical trials help provide invaluable information to empower healthcare professionals to treat, diagnose, or discover new breakthroughs in treating disease. By choosing to participate in a clinical trial, people may help advance treatments, provide crucial insights, and ultimately improve the quality of life for others. A person might also benefit in learning more about their condition, gain access to specific care, or achieve faster access to treatment.

Sponsored by Ultragenyx