Approved Therapies for Duchenne Muscular Dystrophy

Drug Name	Mechanism of Action	Age Criteria	Genetic Variant Criteria	Administration & Frequency
ELEVIDYS (delandistrogene moxeparvovec-rokl)	Restoring or replacing dystrophin	4 years and older who are ambulatory *	Contraindicated for deletions of exon 8 & 9	1 time IV infusion
AMONDYS 45 (casimersen)	Restoring or replacing dystrophin	n/a	Exon 45 skip amenable	1x/week IV infusion
EXONDYS 51 (eteplirsen)	Restoring or replacing dystrophin	n/a	Exon 51 skip amenable	1x/week IV infusion
VILTEPSO (viltolarsen)	Restoring or replacing dystrophin	n/a	Exon 53 skip amenable	1x/week IV infusion
VYONDYS 53 (golodirsen)	Restoring or replacing dystrophin	n/a	Exon 53 skip amenable	1x/week IV infusion
AGAMREE (vamorolone)	Reducing inflammation	2 years and older	none	Oral once daily
EMFLAZA (deflazacort)	Reducing inflammation	2 years and older	none	Oral once daily
DUVYZAT (givinostat)	Improving muscle growth & protection	6 years and older	none	Oral twice daily

ELEVIDYS (delandistrogene moxeparvovec-rokl)

ELEVIDYS (delandistrogene moxeparvovec-rokl) is a one time gene therapy for individuals at least 4 years of age with Duchenne who are ambulatory. It delivers a “micro-dystrophin” gene through an AAV virus to help protect & stabilize muscle function
- Learn more about gene therapies in PPMD’s Gene Therapy Hub
ELEVIDYS is given as a one-time infusion into the vein and requires close follow up and monitoring from your neuromuscular team
Individuals with any deletion in exon 8 and/or exon 9 in the DMD gene will not be able to receive ELEVIDYS due to safety concerns.

Learn more about how ELEVIDYS works, side effects, precautions & safety monitoring and more here.

AMONDYS 45 (casimersen)

AMONDYS 45 (casimersen) is for individuals with Duchenne muscular dystrophy who have a specific genetic variant amenable to exon 45 skipping.
- If you know your/your child’s genetic change (variant) is an exon deletion, PPMD’s Exon Deletion Tool can help you understand if you/your child may be a candidate for AMONDYS 45.
AMONDYS 45 is a once-weekly infusion, usually through an IV (intravenous) or a port.
It helps the body make a shorter but functional dystrophin protein, which aims to slow down muscle damage and progression.

Learn more about AMONDYS 45 side effects, how to access & more here.

EXONDYS 51 (eteplirsen)

EXONDYS 51 (eteplirsen) is for individuals with Duchenne muscular dystrophy who have a specific genetic variant amenable to exon 51 skipping
EXONDYS 51 is a once-weekly infusion, usually through an IV (intravenous) or a port.
It helps the body make a shorter but functional dystrophin protein, which aims to slow down muscle damage and progression.

Learn more about EXONDYS 51 side effects, how to access & more here.

VILTEPSO (viltolarsen)

VILTEPSO™ (viltolarsen) is for individuals with Duchenne muscular dystrophy who have a specific genetic variant amenable to exon 53 skipping.
VILTEPSO is a once-weekly infusion, usually through an IV (intravenous) or a port.
It helps the body make a shorter but functional dystrophin protein, which aims to slow down muscle damage and progression.

Learn more about VILTEPSO side effects, how to access & more here.

VYONDYS 53 (golodirsen)

VYONDYS 53 (golodirsen)is for individuals with Duchenne muscular dystrophy who have a specific genetic variant amenable to exon 53 skipping.
VYONDYS 53 is a once-weekly infusion, usually through an IV (intravenous) or a port.
It helps the body make a shorter but functional dystrophin protein, which aims to slow down muscle damage and progression.
Learn more about VYONDYS 53 side effects, how to access & more here.

AGAMREE (vamorolone)

AGAMREE (vamorolone) is a daily oral medication used to help people ages 2 years and older with Duchenne. It belongs to a group of medicines called corticosteroids, which help reduce inflammation and support muscle strength.
AGAMREE is not dependent upon genetic variants or other factors like ambulation.
You should never stop taking AGAMREE abruptly and always consult with your doctor if thinking about stopping or switching.
AGAMREE is designed to keep the helpful effects of traditional steroids while aiming to lower the chance of common side effects like weaker bones, slower growth, mood changes, and delayed puberty.

Learn more about AGAMREE side effects, precautions, & how to access here.

EMFLAZA (deflazacort)

EMFLAZA (deflazacort) is an oral corticosteroid that reduces muscle inflammation and slows muscle weakness in individuals with Duchenne. EMFLAZA is the brand name and deflazacort is the generic name.
EMFLAZA (deflazacort) is not dependent upon genetic variants or other factors like ambulation and is available for individuals ages 2 years and older.
You should never stop taking EMFLAZA (deflazacort) abruptly and always consult with your doctor if thinking about stopping or switching.

Learn more about EMFLAZA (deflazacort) side effects, precautions, & how to access here.

DUVYZAT (givinostat)

DUVYZAT™ (givinostat) is an ‘HDAC inhibitor’ which helps the body repair muscles, reduce inflammation, and slow down muscle loss. DUVYZAT is not a steroid.
DUVYZAT is approved for individuals with Duchenne ages 6 years and older, no matter what genetic variant they have.
Duvyzat is a liquid taken by mouth twice a day with food. Some kids may have side effects like GI (digestive) issues or low platelets, so doctors will monitor closely.

Learn more about DUVYZAT side effects, precautions, monitoring, & how to access here.

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