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Downloads
Download GENGLYCOS materials
GENGLYCOS Patient Brochure
Learn about GENGLYCOS, including how it works and the results of the clinical studies.
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GENGLYCOS Treatment Roadmap
Understand important steps in the treatment process, so you know what to expect and how to prepare for your GENGLYCOS infusion.
Download NowUltraCare® Gene Therapy
Ultragenyx is committed to supporting individuals with rare diseases, which is why we created UltraCare Gene Therapy Guides. We are here to guide you and your family through every step of the GENGLYCOS treatment journey.
UltraCare Patient Services for Gene Therapy will help you:
Understand insurance coverage
Determine your eligibility for financial assistance
Navigate travel logistics
Access available resources
UltraCare will also support you and your family with understanding the Qualified Treatment Center’s individualized posttreatment plan.
Work with your doctor to enroll in UltraCare Patient Services for Gene Therapy by completing the enrollment form together and then having your doctor submit it to UltraCare.
If you are already enrolled in UltraCare, call your UltraCare Gene Therapy Guide today at 1-888-756-8657, option 1. They can tailor their assistance to your unique needs.
To learn more, visit UltraCareSupport.com.
Advocacy Groups*
Connect with the glycogen storage disease type Ia (GSDIa) and rare disease communities
Association for Glycogen Storage Disease (AGSD)
AGSD is a parent- and patient-oriented support group that is advised by a group of experienced medical professionals.
The Children’s Fund for Glycogen Storage Disease Research
The Children’s Fund for Glycogen Storage Disease Research is a
public not-for-profit 501(c)(3) foundation that aims to make a difference in the lives of
children and their families affected by GSDI.
Global Center for Glycogen Storage Disease (GSD)
The Global Center for GSD seeks to ensure the sharing of information from different parties about this rare disease. This includes researchers, doctors, and people afflicted with this disease.
Global Genes®: Allies in Rare Disease
Global Genes is a 501(c)(3) nonprofit organization dedicated to
eliminating the burdens and challenges of rare diseases for patients and families
globally.
EveryLife Foundation for Rare Diseases
The EveryLife Foundation for Rare Diseases is a 501(c)(3)
nonprofit, nonpartisan organization dedicated to empowering the rare disease patient
community.
National Organization for Rare Disorders (NORD)
NORD advances practical, meaningful, and enduring change so people with rare diseases can live their fullest and best lives.
*Inclusion of these links is not intended as an endorsement of these organizations or the services they may offer. The organizations listed are responsible for the content or services described on their websites.
Frequently Asked Questions
Get answers to frequently asked questions about GENGLYCOS
What is GENGLYCOS?
GENGLYCOS is the first and only FDA-approved gene therapy for individuals 8 years and older with GSDIa. It is a one-time intravenous (IV) infusion and is used to reduce daily cornstarch intake along with nutritional management. GENGLYCOS is designed to deliver a working copy of the G6PC gene into the body to target GSDIa at the genetic level.
Who is eligible for treatment with GENGLYCOS?
GENGLYCOS is approved for individuals 8 years and older with GSDIa to reduce daily cornstarch intake along with nutritional management. Some individuals may not be eligible to receive GENGLYCOS, including those who have certain forms of liver damage such as fibrosis or cirrhosis, and those who have pre-existing antibodies to adeno-associated virus serotype 8 (AAV8). If you are considering GENGLYCOS, talk with your doctor about which tests you might need to find out if you’re eligible.
Learn more about eligibility and the GENGLYCOS treatment journey
How might GENGLYCOS help address my GSDIa management goals?
In a clinical study, those who received GENGLYCOS were able to reduce their average daily cornstarch intake versus placebo at 48 weeks. Average reductions were seen for both the amount of daily cornstarch needed and the overall number of daily doses needed.
How will my nutritional needs and diet change after GENGLYCOS?
As your body adjusts to treatment with GENGLYCOS, you and your doctor may be able to work together to make changes to your nutritional goals. This could include reducing the amount of cornstarch you consume each day, making modifications to your meal plan, or changing the vitamin or mineral supplements you take.
You’ll need to work closely with your care team on any nutritional changes and continue monitoring your blood glucose levels to understand how you are responding to any new changes.
What are the possible or likely side effects of GENGLYCOS?
Seven serious side effects were observed in a GENGLYCOS study, including anaphylaxis/infusion reaction (2), adrenal gland problems (2), high lactate level (2), and low blood sugar (1).
The most common side effects that occurred in individuals treated with GENGLYCOS in a study were:
- Increased liver enzymes
- Nausea
- Headache
- High triglycerides (high levels of a type of fat in the blood)
- Low adrenal hormone levels or adrenal gland problems
- Constipation
- High blood sugar
- Acne or acne-like skin rash
- Cushingoid features (changes caused by high steroid levels such as rounded face, weight gain, or increased body fat)
- Anaphylaxis (severe allergic reaction)
These are not all the possible side effects of treatment with GENGLYCOS.
Learn more about the safety seen in GENGLYCOS clinical studies
What is the GENGLYCOS infusion process like?
GENGLYCOS is a one-time intravenous (IV) infusion that takes around 3 hours.
Where can I receive a GENGLYCOS infusion?
GENGLYCOS must be administered at a Qualified Treatment Center (QTC). These are independently owned and operated facilities with staff who have been trained to administer GENGLYCOS gene therapy. Your doctor can work with UltraCare support services to identify potential QTCs for the infusion.
What is UltraCare Patient Services for Gene Therapy?
UltraCare Patient Services for Gene Therapy is a service provided by Ultragenyx that’s designed to support you at every step of the treatment journey. Once enrolled in UltraCare, you will work with a Gene Therapy Guide who can help with:
- Understanding insurance coverage
- Determining eligibility for financial assistance
- Navigating travel logistics
- Providing access to educational resources
Learn more about GENGLYCOS.
See the Study ResultsWHAT IS GENGLYCOS
(pariglasgene brecaparvovec-opnr)?
GENGLYCOS is a prescription gene therapy used along with nutritional management to help reduce the amount of cornstarch taken daily in adults and children 8 years and older with Glycogen Storage Disease type Ia (GSDla).
This use is approved under accelerated approval based on clinical study results that measured reduction in daily cornstarch intake. Verification of a clinical benefit may be needed for continued approval of GENGLYCOS.
IMPORTANT SAFETY INFORMATION
Do not use GENGLYCOS if you have severe liver scarring (fibrosis or cirrhosis).
What is the most important information I should know about GENGLYCOS?
- Allergic and infusion reactions, including anaphylaxis, occurred with GENGLYCOS treatment. Some of these reactions have been severe. You will be closely monitored during and after infusion for signs and symptoms including hives, flushing, low blood pressure, wheezing, trouble breathing, chest tightness, nausea, vomiting, headache, stomach pain, lightheadedness, flu-like symptoms, shivering, rash, and high blood pressure. If you think you’re having a reaction to the infusion, contact your healthcare provider immediately.
- GENGLYCOS can cause certain liver enzymes to increase. You will receive oral corticosteroids with or without other medications after your infusion and will receive regular blood tests to monitor your liver function. Contact your healthcare provider right away if you experience unusual tiredness, yellowing of your skin and/or eyes, dark urine, nausea, vomiting, or pain in the upper right side of your stomach area, or if you miss a dose of your corticosteroid or vomit after taking it.
- Do not take any medicines, including herbal products or nutritional supplements, before confirming with your healthcare provider that they are not harmful to the liver.
- Talk to your healthcare provider about the possible risks of drinking alcohol following GENGLYCOS infusion.
- Problems with adrenal glands, including serious events, have occurred in patients receiving GENGLYCOS during corticosteroid treatment. Signs of adrenal gland problems can include unusual tiredness, weakness, loss of appetite, nausea, vomiting, low blood pressure, low sodium levels in the blood, and low blood sugar. A severe form called adrenal crisis can cause very low blood pressure, sudden severe stomach pain, or loss of consciousness. You will be monitored for signs and symptoms of adrenal gland problems. Do not stop taking your corticosteroid suddenly—follow your provider’s instructions.
- There is a theoretical risk of tumor growth with gene therapies like GENGLYCOS; no cases have been reported in GENGLYCOS clinical studies. Contact your healthcare provider and Ultragenyx Pharmaceutical Inc. at 1-888-756-86571-888-756-8657 if a tumor develops.
Are there any considerations for vaccination schedules and GENGLYCOS?
- Talk to your healthcare provider to determine if your vaccination schedule needs to be adjusted to accommodate corticosteroid and other medication administration prior to and after GENGLYCOS infusion.
- Vaccines should be avoided 1 month prior to GENGLYCOS administration.
What do I need to know about pregnancy and breastfeeding with GENGLYCOS?
- It is not known if GENGLYCOS can harm your baby or affect your ability to become pregnant. GENGLYCOS should not be used during pregnancy.
- It is not known if GENGLYCOS passes into breast milk. Talk to your healthcare provider about the best way to feed your baby if you take GENGLYCOS.
What do I need to know about contraception and sperm donation with GENGLYCOS?
- Women of childbearing potential should use an effective method of contraception for at least 12 months after administration of GENGLYCOS.
- For 6 months after GENGLYCOS administration, men should not donate sperm, and men capable of fathering a child and their female partner should avoid pregnancy and use an effective form of birth control.
Are there any precautions that need to be considered when handling a patient’s bodily waste?
- Small amounts of GENGLYCOS can travel through the patient’s blood and be temporarily passed through urine, stool, and saliva. Talk to your healthcare provider about proper hygiene when handling patient body waste. Precautions should be followed for 3 months after GENGLYCOS infusion.
What are the possible or likely side effects of GENGLYCOS?
Seven serious side effects were observed in a GENGLYCOS study, including anaphylaxis/infusion reaction (2), adrenal gland problems (2), high lactate level (2), and low blood sugar (1).
The most common side effects that occurred in individuals treated with GENGLYCOS in a study were:
- Increased liver enzymes
- Nausea
- Headache
- High triglycerides (high levels of a type of fat in the blood)
- Low adrenal hormone levels or adrenal gland problems
- Constipation
- High blood sugar
- Acne or acne-like skin rash
- Cushingoid features (changes caused by high steroid levels such as rounded face, weight gain, or increased body fat)
- Anaphylaxis (severe allergic reaction)
These are not all the possible side effects of GENGLYCOS. Talk to your healthcare provider about any side effects that bother you or don’t go away.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657.