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Medications for Mucopolysaccharidosis Type VII

Other names: GUSB Deficiency; MPS VII; Sly Syndrome

About Mucopolysaccharidosis Type VII

Mucopolysaccharidosis VII (also known as Sly Syndrome, MPS VII and GUSB Deficiency) is a rare genetic disorder caused by the lack of the enzyme beta-glucuronidase, which breaks down the long chain sugar molecule called glycosaminoglycans (GAG).

Usually GAG is broken down by the enzyme beta-glucuronidase into simpler molecules that the cell can reuse.  In MPS VII there is not enough enzyme produced, or the enzymes do not work properly, so the GAG is not broken down and therefore starts to accumulate within the cells. This build up of GAG causes progressive, permanent damage to the cells which produces the symptoms and physical problems of MPS VII which can range from mild to severe.

Symptoms and physical problems include

  • skeletal irregularities
  • short stature
  • joint stiffness
  • eye issues including corneal clouding, loss of peripheral vision and night blindness
  • hearing problems
  • developmental delay and a progressive decline developmental function
  • respiratory issues
  • enlarged liver and spleen
  • heart problems

Drugs used to treat Mucopolysaccharidosis Type VII

The following list of medications are in some way related to, or used in the treatment of this condition.

Drug name Rating Reviews Activity ? Rx/OTC Pregnancy CSA Alcohol
Mepsevii Rate Add review
Rx N

Generic name: vestronidase alfa systemic

Drug class: lysosomal enzymes

For consumers: dosage, side effects

For professionals: Prescribing Information

vestronidase alfa Rate Add review
Rx N

Generic name: vestronidase alfa systemic

Brand name:  Mepsevii

Drug class: lysosomal enzymes

For consumers: dosage, side effects

For professionals: A-Z Drug Facts, AHFS DI Monograph

Legend

Rating For ratings, users were asked how effective they found the medicine while considering positive/adverse effects and ease of use (1 = not effective, 10 = most effective).
Activity Activity is based on recent site visitor activity relative to other medications in the list.
Rx Prescription Only.
OTC Over the Counter.
Rx/OTC Prescription or Over the Counter.
Off-label This medication may not be approved by the FDA for the treatment of this condition.
Pregnancy Category
A Adequate and well-controlled studies have failed to demonstrate a risk to the fetus in the first trimester of pregnancy (and there is no evidence of risk in later trimesters).
B Animal reproduction studies have failed to demonstrate a risk to the fetus and there are no adequate and well-controlled studies in pregnant women.
C Animal reproduction studies have shown an adverse effect on the fetus and there are no adequate and well-controlled studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
D There is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
X Studies in animals or humans have demonstrated fetal abnormalities and/or there is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience, and the risks involved in use in pregnant women clearly outweigh potential benefits.
N FDA has not classified the drug.
Controlled Substances Act (CSA) Schedule
N Is not subject to the Controlled Substances Act.
1 Has a high potential for abuse. Has no currently accepted medical use in treatment in the United States. There is a lack of accepted safety for use under medical supervision.
2 Has a high potential for abuse. Has a currently accepted medical use in treatment in the United States or a currently accepted medical use with severe restrictions. Abuse may lead to severe psychological or physical dependence.
3 Has a potential for abuse less than those in schedules 1 and 2. Has a currently accepted medical use in treatment in the United States. Abuse may lead to moderate or low physical dependence or high psychological dependence.
4 Has a low potential for abuse relative to those in schedule 3. It has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 3.
5 Has a low potential for abuse relative to those in schedule 4. Has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 4.
Alcohol
X Interacts with Alcohol.

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Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.