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Medications for Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy is the most common childhood form of muscular dystrophy and is caused by a mutation in the dystrophin gene.

Drugs used to treat Duchenne Muscular Dystrophy

The medications listed below are related to or used in the treatment of this condition.

Drug name Rating Reviews Activity ? Rx/OTC Pregnancy CSA Alcohol
deflazacort Rate Add review Rx N
Generic name:
deflazacort systemic
Brand name:
Emflaza
Drug class:
glucocorticoids
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph, Prescribing Information
Exondys 51 Rate Add review Rx N
Generic name:
eteplirsen systemic
Drug class:
miscellaneous uncategorized agents
For consumers:
dosage, side effects
For professionals:
Prescribing Information
Emflaza Rate Add review Rx N
Generic name:
deflazacort systemic
Drug class:
glucocorticoids
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
Viltepso Rate Add review Rx N
Generic name:
viltolarsen systemic
Drug class:
miscellaneous uncategorized agents
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
viltolarsen Rate Add review Rx N
Generic name:
viltolarsen systemic
Brand name:
Viltepso
Drug class:
miscellaneous uncategorized agents
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph
delandistrogene moxeparvovec Rate Add review Rx N
Generic name:
delandistrogene moxeparvovec systemic
Brand name:
Elevidys
Drug class:
miscellaneous uncategorized agents
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph
Elevidys Rate Add review Rx N
Generic name:
delandistrogene moxeparvovec systemic
Drug class:
miscellaneous uncategorized agents
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
Duvyzat Rate Add review Rx N
Generic name:
givinostat systemic
Drug class:
histone deacetylase inhibitors
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
givinostat Rate Add review Rx N
Generic name:
givinostat systemic
Brand name:
Duvyzat
Drug class:
histone deacetylase inhibitors
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph
Amondys 45 Rate Add review Rx N
Generic name:
casimersen systemic
Drug class:
miscellaneous uncategorized agents
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
eteplirsen Rate Add review Rx N
Generic name:
eteplirsen systemic
Brand name:
Exondys 51
Drug class:
miscellaneous uncategorized agents
For consumers:
dosage, side effects
For professionals:
AHFS DI Monograph
Vyondys 53 Rate Add review Rx N
Generic name:
golodirsen systemic
Drug class:
miscellaneous uncategorized agents
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
golodirsen Rate Add review Rx N
Generic name:
golodirsen systemic
Brand name:
Vyondys 53
Drug class:
miscellaneous uncategorized agents
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph
casimersen 10 1 review for casimersen to treat Duchenne Muscular Dystrophy Rx N
Generic name:
casimersen systemic
Brand name:
Amondys 45
Drug class:
miscellaneous uncategorized agents
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph
Agamree Rate Add review Rx N
Generic name:
vamorolone systemic
Drug class:
glucocorticoids
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
vamorolone Rate Add review Rx N
Generic name:
vamorolone systemic
Brand name:
Agamree
Drug class:
glucocorticoids
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph

Frequently asked questions

View more FAQ

Learn more about Duchenne Muscular Dystrophy

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.
EUA An Emergency Use Authorization (EUA) allows the FDA to authorize unapproved medical products or unapproved uses of approved medical products to be used in a declared public health emergency when there are no adequate, approved, and available alternatives.
Expanded Access Expanded Access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
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
M The drug has multiple schedules. The schedule may depend on the exact dosage form or strength of the medication.
U CSA Schedule is unknown.
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.

Further information

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