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Pompe Disease
Medications for Pompe disease
About Pompe disease: Glycogen storage disease type II (also known as Pompe disease or acid maltase deficiency) is an neuromuscular, autosomal recessive metabolic disorder from the category of lysosomal storage diseases brought on by a deficiency in the enzyme Acid alpha-glucosidase (EC 3.2.1.20), that will be required to break down glycogen--a lengthy, branched glucose polymer and kept form of sugar used for energy. It`s the sole glycogen storage disorder with a flaw in metabolic process and has been the first glycogen storage disorder to be identified, in 1932.
The accumulation of glycogen causes progressive muscle weakness (myopathy) throughout the body and affects many different body cells, especially in the heart, skeletal muscles, liver and nervous system.
The disorder is named after Johann Pompe, who distinguished it at 1932.
Drugs Used to Deal with Pompe disease
This list of medications are somehow associated with, or found at the treating this illness.
| Medication title | Rx / OTC | Pregnancy | CSA | Alcohol | Reviews | Rating | Popularity |
|---|---|---|---|---|---|---|---|
| Lumizyme | Rx | B | N | Add inspection | 1.0 | ||
Generic name: alglucosidase alfa systemic Drug category: lysosomal enzymes For users: dose, side effects For specialists: Prescribing Information | |||||||
| alglucosidase alfa | Rx | B | N | Add inspection | 1.0 | ||
Generic name: alglucosidase alfa systemic Brand titles: Lumizyme, Myozyme Drug category: lysosomal enzymes For users: dosage, For specialists: A-Z Drug Truth, AHFS DI Monograph | |||||||
| Myozyme | Rx | B | N | Add inspection | 0.0 | ||
Generic name: alglucosidase alfa systemic Drug category: lysosomal enzymes For users: dose, side effects For professionals: AHFS DI Monograph, Prescribing Information | |||||||
Legend
| Rx | prescription-only |
|---|---|
| OTC | On The Counter |
| Rx/OTC | Prescription or Over the Counter |
| Away Tag | This medication may not be approved by the FDA for treating the illness. |
| Pregnancy Category | |
|---|---|
| A | Adequate and well-controlled studies have failed to present a risk to the embryo in the first trimester of pregnancy (and there isn`t any evidence of risk in later trimesters). |
| B | Animal reproduction studies have failed to show a risk to the fetus and there are no adequate and well-controlled studies in pregnant women. |
| C | Animal reproduction studies have demonstrated a negative effect in the fetus and there are no adequate and well-controlled studies in humans, but possible 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 possible benefits may warrant use in pregnant women despite potential risks. |
| X | Studies in animals or humans have demonstrated fetal abnormalities and/or there`s positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience, and also the risks involved with use in pregnant women obviously outweigh potential rewards. |
| N | FDA have not classified this medication. |
| Controlled Substances Act (CSA) Schedule | |
|---|---|
| N | isn`t subject to the Controlled Substances Act. |
| Inch | features a higher potential for abuse. Has no currently accepted medical use in treatment in the United States. There`s a lack of accepted safety for use under medical supervision. |
| 2 | features a higher potential for abuse. Includes a currently accepted medical use with severe restrictions or a currently accepted medical use in treatment in the United States. Abuse may lead to severe psychological or physical dependency. |
| 3 | includes a possibility for abuse less than individuals in programs 1 and two. Includes 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 | features a minimal potential for abuse in accordance with individuals in program 3. It`s a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to individuals in program 3. |
| 5 | features a minimal potential for abuse in accordance with individuals in program 4. Includes a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence. |
| Alcohol | |
|---|---|
| X | Interacts with Alcohol. |
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Always ask your healthcare provider to make sure the information displayed on these pages relates to your personal circumstances.
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