Treatment & Care

At the many Duchenne Certified Care Centers in the United States, you will find the top Duchenne experts in the field, the current standards of care guidelines, and the most promising clinical trial options available.

Approved Treatments

Explore the variety of approved Duchenne treatments below.

FDA Approved Drugs

Types of Mutations

Duchenne is caused by mutations (changes) within the dystrophin gene. A gene is made up of coding regions called exons, and the areas in between exons are called introns. Dystrophin has 79 exons, which makes it one of the largest genes in the body.

Making the dystrophin protein from the gene involves several steps. One of the first steps is removing the introns and fitting the exons together, 1-79, like puzzle pieces.

If there is a missing piece within the dystrophin gene (deletion) or an extra piece (duplication), your body can have difficulty making dystrophin.

Visit Parent Project Muscular Dystrophy below for further resources about Duchenne testing and mutations.

Quick Links

Use the resources on the right to learn more about genetics and information surrounding mutations and testing.

Insurance Flight Plan

Navigating insurance can be a tricky task, especially when you or your child requires specialized care and equipment. Our Health Insurance Flight Plan breaks down options that may be available to you and provides a launching ground for families as they begin to think about what plan may be best for them.

The booklet is free of charge and available to download online. Fill out the form below and your booklet will be on its way!

Download Health Insurance Flight Plan

Company	Drug Name	Drug Type	Route of Administration	Exons Eligible	Age	Ambulation Status
Aucta Pharmaceuticals, Inc.	PYQUVI ™ (deflazacort)	Corticosteroid	Oral	Mutation Agnostic – All eligible	5 years of age and older	Ambulatory and Non-Ambulatory
Catalyst Pharmceuticals	aGamree (Vamorolone)	Corticosteroid	Oral	Mutation Agnostic – All eligible	2 years of age and older	Ambulatory and Non-Ambulatory
Cranbury Pharmaceuticals, LLC	Deflazacort Oral Suspension	Corticosteroid	Oral	Mutation Agnostic – All eligible	5 years of age and older	Ambulatory and Non-Ambulatory
ITF Therapeutics	Duvyzat (givinostat)	HDAC Inhibitor	Oral	Mutation Agnostic – All eligible	6 years of age and older	Ambulatory and Non-Ambulatory
NS Pharma	Viltepso (vitolarsen)	Exon Skipping 53	IV Infusion	Please see Exon Deletion Tool or Chart for more information*	All ages	Ambulatory and Non-Ambulatory
PTC Therapeutics	Emflaza (deflazacort)	Corticosteroid	Oral	Mutation Agnostic – All eligible	2 years of age and older	Ambulatory and Non-Ambulatory
Sarepta Therapeutics	Exondys 51 (eteplirsen)	Exon Skipping 51	IV Infusion	Refer to exon skipping tool for more information.	All ages	Ambulatory and Non-Ambulatory
Sarepta Therapeutics	Elevidys (delandistrogene moxeparvovec-rokl)	Gene Therapy	IV Infusion	All mutations except those with any deletion in exon 8 and/or exon 9 in the DMD gene	At least 4 years of age	Ambulatory
Sarepta Therapeutics	Amondys 45 (casimersen)	Exon Skipping 45	IV Infusion	Refer to exon skipping tool for more information.	All ages	Ambulatory and Non-Ambulatory
Sarepta Therapeutics	Vyondys 53 (golodirsen)	Exon Skipping 53	IV Infusion	Refer to exon skipping tool for more information.	All ages	Ambulatory and Non-Ambulatory
Upsher-Smith	Kymbee (deflazacort)	Corticosteroid	Oral	Mutation Agnostic – All eligible	5 years of age and older	Ambulatory and Non-Ambulatory
Zydus	Jaythari (deflazacort)	Corticosteroid	Oral	Mutation Agnostic – All eligible	5 years of age and older	Ambulatory and Non-Ambulatory

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