Pariglasgene Brecaparvovec

This information from UpToDate^® Lexidrug^™ explains what you need to know about this medication, including what it’s used for, how to take it, its side effects, and when to call your healthcare provider.

Brand Names: US

Genglycos >87kg; Genglycos 15 to <21kg; Genglycos 21 to <27kg; Genglycos 27 to <33kg; Genglycos 33 to <39kg; Genglycos 39 to <45kg; Genglycos 45 to <51kg; Genglycos 51 to <57kg; Genglycos 57 to <63kg; Genglycos 63 to <69kg; Genglycos 69 to <75kg; Genglycos 75 to <81kg; Genglycos 81 to <87kg

What is this drug used for?

• It is used to help lower daily cornstarch intake in people with glycogen storage disease type 1a (GSD1a).

What do I need to tell the doctor BEFORE my child takes this drug?

• If your child is allergic to this drug; any part of this drug; or any other drugs, foods, or substances. Tell the doctor about the allergy and what signs your child had.
• If your child has antibodies to AAV8.
• If your child has a liver problem, including liver infection.
• If your child has had a vaccine in the past month.
• If your child is pregnant or may be pregnant. Do not give this drug to your child during pregnancy.

This is not a list of all drugs or health problems that interact with this drug.

Tell the doctor and pharmacist about all of your child’s drugs (prescription or OTC, natural products, vitamins) and health problems. You must check to make sure that it is safe to give this drug with all of your child’s other drugs and health problems. Do not start, stop, or change the dose of any drug your child takes without checking with the doctor.

What are some things I need to know or do while my child takes this drug?

• Tell all of your child’s health care providers that your child is taking this drug. This includes your child’s doctors, nurses, pharmacists, and dentists.
• Have your child’s blood work and other lab tests checked as you have been told by the doctor.
• High triglyceride levels have happened with this drug. Tell your child’s doctor if your child has ever had high triglyceride levels.
• Talk with the doctor before getting your child any vaccines. Use of some vaccines with this drug may either raise the chance of an infection or make the vaccine not work as well.
• This drug may be passed to others through body fluids after the infusion. This happens through blood, saliva, urine, stools, and semen. Talk with the doctor about how to avoid spreading this drug to others. Be sure to wash hands well and dispose of anything that comes into contact with body fluids as you have been told. You will need to do this for 3 months after your child gets this drug.
• There is a chance that this drug could raise the risk of a tumor. Talk with the doctor if you have questions.
• Be sure your child does not donate semen for at least 6 months after getting this drug.

If your child is or may be sexually active:

• If your child may become pregnant, birth control must be used for at least 12 months after your child gets this drug. If your child gets pregnant, call the doctor right away.
• If your child’s sex partner may become pregnant, birth control must be used for 6 months after your child gets this drug. If your child’s partner gets pregnant, call the doctor right away.

If your child is breast-feeding a baby:

• Tell the doctor if your child is breast-feeding a baby. You will need to talk about any risks to the baby.

What are some side effects that I need to call my child’s doctor about right away?

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your child’s doctor or get medical help right away if your child has any of the following signs or symptoms that may be related to a very bad side effect:

• Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.
• Signs of liver problems like dark urine, tiredness, decreased appetite, upset stomach or stomach pain, light-colored stools, throwing up, or yellow skin or eyes.
• Signs of a weak adrenal gland like a severe upset stomach or throwing up, severe dizziness or passing out, muscle weakness, feeling very tired, mood changes, decreased appetite, or weight loss.
• Signs of Cushing’s syndrome like weight gain in the upper back or belly, moon face, severe headache, or slow healing.
• Signs of high blood sugar like confusion, feeling sleepy, unusual thirst or hunger, passing urine more often, flushing, fast breathing, or breath that smells like fruit.
• Infusion reactions have happened with this drug. Sometimes these can be severe. Tell the doctor right away if your child has signs of high or low blood pressure like headache, dizziness, passing out, or change in eyesight. Tell the doctor right away if your child has fever, chills, flushing, flu-like signs, trouble breathing, chest tightness, upset stomach, throwing up, stomach pain, or any other bad effect while this drug is being given.

What are some other side effects of this drug?

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your child’s doctor or get medical help if any of these side effects or any other side effects bother your child or do not go away:

• Pimples (acne).
• Constipation.
• Headache and upset stomach are common with this drug. However, if they happen during the infusion, they may be part of an infusion reaction. Tell the doctor if your child has a headache or upset stomach while this drug is being given.

These are not all of the side effects that may occur. If you have questions about side effects, call your child’s doctor. Call your child’s doctor for medical advice about side effects.

You may report side effects to your national health agency.

How is this drug best given?

Give this drug as ordered by your child’s doctor. Read all information given to you. Follow all instructions closely.

• It is given as an infusion into a vein over a period of time.
• Other drugs will be given with this drug to help avoid side effects.

What do I do if my child misses a dose?

• Call your child’s doctor to find out what to do.

How do I store and/or throw out this drug?

• If you need to store this drug at home, talk with your child’s doctor, nurse, or pharmacist about how to store it.

General drug facts

• If your child’s symptoms or health problems do not get better or if they become worse, call your child’s doctor.
• Do not share your child’s drug with others and do not give anyone else’s drug to your child.
• Keep all drugs in a safe place. Keep all drugs out of the reach of children and pets.
• Throw away unused or expired drugs. Do not flush down a toilet or pour down a drain unless you are told to do so. Check with your pharmacist if you have questions about the best way to throw out drugs. There may be drug take-back programs in your area.
• Some drugs may have another patient information leaflet. If you have any questions about this drug, please talk with your child’s doctor, nurse, pharmacist, or other health care provider.
• If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.

Consumer Information Use and Disclaimer

This generalized information is a limited summary of diagnosis, treatment, and/or medication information. It is not meant to be comprehensive and should be used as a tool to help the user understand and/or assess potential diagnostic and treatment options. It does NOT include all information about conditions, treatments, medications, side effects, or risks that may apply to a specific patient. It is not intended to be medical advice or a substitute for the medical advice, diagnosis, or treatment of a health care provider based on the health care provider’s examination and assessment of a patient’s specific and unique circumstances. Patients must speak with a health care provider for complete information about their health, medical questions, and treatment options, including any risks or benefits regarding use of medications. This information does not endorse any treatments or medications as safe, effective, or approved for treating a specific patient. UpToDate, Inc. and its affiliates disclaim any warranty or liability relating to this information or the use thereof. The use of this information is governed by the Terms of Use, available at https://www.wolterskluwer.com/en/know/clinical-effectiveness-terms.

Last Reviewed Date

2026-08-27

Copyright

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