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Generic Name: laronidase (lar ON i dase)
Medically reviewed by Holevn.org on Dec 11, 2019 – Written by Cerner Multum
What is laronidase?
Laronidase contains an enzyme that occurs naturally in the body in healthy people. Some people lack this enzyme because of a genetic disorder. Laronidase helps replace this missing enzyme in such people.
Laronidase is used to treat some of the symptoms of a genetic condition called mucopolysaccharidosis (MYOO-koe-pol-ee-SAK-a-rye-DOE-sis) or MPS I, also called Hurler syndrome, Hurler-Scheie syndrome, or Scheie syndrome.
MPS I is a metabolic disorder in which the body lacks the enzyme needed to break down certain substances. These substances can build up in the body, causing enlarged organs, abnormal bone structure, changes in facial features, breathing problems, heart problems, vision or hearing loss, and changes in mental or physical abilities.
Laronidase may improve breathing and walking ability in people with this condition. However, this medication is not a cure for MPS I.
Laronidase may also be used for purposes not listed in this medication guide.
An allergic reaction may occur during or shortly after infusion of laronidase. Tell your caregivers or get emergency medical help right away if you have any signs of a severe allergic reaction, such as sweating, chest discomfort, trouble breathing, pale skin, blue lips or fingernails, and feeling like you might pass out.
Before taking this medicine
Your laronidase infusion may be delayed if you have a fever or cold symptoms.
Tell your doctor if you have ever had:
heart disease; or
sleep apnea, and you use a continuous positive airway pressure (CPAP) machine.
Tell your doctor if you are pregnant or breastfeeding.
It is not known whether laronidase will harm an unborn baby. However, MPS symptoms can get worse during pregnancy and may cause medical problems in both mother and baby. The benefit of treating MPS may outweigh any risks to the baby.
Your name may need to be listed on an MPS I Registry while you are using this medicine. The purpose of this registry is to track the progression of this disorder and the effects that laronidase has on long-term treatment of MPS I. The registry also tracks the effects of laronidase on a baby if you are pregnant or breastfeeding.
How is laronidase given?
Laronidase is given as an infusion into a vein. A healthcare provider will give you this injection.
Laronidase is usually given once per week.
Tell your doctor if you have been sick with a fever or cold. You may need to wait until you get better before receiving your dose of laronidase.
Laronidase must be given slowly, and the infusion can take up to 4 hours to complete.
An hour before each injection, you will be given other medications to help prevent a serious allergic reaction.
After each injection, you will be watched closely to make sure you do not have an allergic reaction.
Your doctor will need to check your progress on a regular basis.
What happens if I miss a dose?
Call your doctor for instructions if you miss an appointment for your laronidase injection.
What happens if I overdose?
Since laronidase is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.
What should I avoid while receiving laronidase?
Follow your doctor’s instructions about any restrictions on food, beverages, or activity.
Laronidase side effects
Get emergency medical help if you have signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.
An allergic reaction may occur during or shortly after infusion of laronidase (up to 3 hours later). Tell your caregivers or get emergency medical help right away if you have any signs of a severe allergic reaction, such as:
rapid breathing, or feeling short of breath;
wheezing, chest tightness;
feeling like you might pass out, even while lying down;
fast or slow heartbeats;
sweating, cold or clammy skin;
pale skin; or
blue lips or fingernails.
Common side effects may include:
cold symptoms such as stuffy nose, sneezing, sore throat;
increased blood pressure;
warmth, redness, numbness, or tingly feeling;
overactive reflexes; or
pain, swelling, burning, or irritation around the IV needle.
This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Laronidase dosing information
Usual Adult Dose for Mucopolysaccharidosis Type I:
0.58 mg/kg IV once a week
-Clinical studies did not include patients 65 years and older and it is therefore unknown if they respond differently than younger patients.
Uses: For the treatment of Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie forms who have moderate to severe symptoms; risk and benefits of treating mildly affected patients with the Scheie form have not been established
Usual Pediatric Dose for Mucopolysaccharidosis Type I:
6 months or older: 0.58 mg/kg IV once a week
Uses: For the treatment of Hurler and Hurler-Scheie forms of MPS I and for patients with the Scheie forms who have moderate to severe symptoms; risk and benefits of treating mildly affected patients with the Scheie form have not been established
What other drugs will affect laronidase?
Other drugs may affect laronidase, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.
Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.
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Reference from: https://www.drugs.com/mtm/laronidase.html