Duchenne and Becker muscular dystrophy

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Who is this for?
Show terms as
4FDA treatments10Specialists8Treatment centers

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Duchenne and Becker Muscular Dystrophy is treated with 3 medications in our database, including Deflazacort, DEFLAZACORT, Vyondys 53, JAYTHARI Oral Suspension. 2 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by PTC Therapeutics, Sarepta, Amneal. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Duchenne and Becker Muscular Dystrophy treatment below.

Also known as:

Inheritance

X-linked recessive

Carried on the X chromosome; typically affects males more than females

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗NORD ↗

FDA & Trial Timeline

1 event
Mar 2024

DUVYZAT: FDA approved

treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older

FDAcompleted

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

4 available

DUVYZAT

GIVINOSTAT· Italfarmaco SPAOrphan Drug

treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older

Deflazacort, DEFLAZACORT

· PTC Therapeutics

* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Not Publicly Available * RxBIN, PCN, and Group numbers: Not Publi

Vyondys 53

golodirsen· Sarepta

* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Not Publicly Available * RxBIN, PCN, and Group numbers: Not Publi

JAYTHARI Oral Suspension

Deflazacort· Amneal

• Patient Copay Amount: As little as $0 per prescription • Maximum Annual Benefit Limit: Not Publicly Available (Maximum value per prescription and annual fill limits apply but specific amounts are no

No actively recruiting trials found for Duchenne and Becker muscular dystrophy at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Duchenne and Becker muscular dystrophy community →

Specialists

10 foundView all specialists →
SO
Satellos Chief Medical Officer
Los Angeles, California
Specialist

Rare Disease Specialist

PI on 1 active trial1 Duchenne and Becker muscular dystrophy publication
LP
Laurent Servais, MD, PhD
Liège
Specialist

Rare Disease Specialist

PI on 2 active trials
DB
Daniel Natera-de Benito
GAINESVILLE, FL
Specialist
2 Duchenne and Becker muscular dystrophy publications
AC
Anna Codina
Specialist
2 Duchenne and Becker muscular dystrophy publications
KM
Katherine D Mathews
Specialist
2 Duchenne and Becker muscular dystrophy publications
JA
Jennifer G Andrews
Specialist
2 Duchenne and Becker muscular dystrophy publications
NW
Nedra Whitehead
NC
Specialist
2 Duchenne and Becker muscular dystrophy publications
ML
Molly M Lamb
CO
Specialist
2 Duchenne and Becker muscular dystrophy publications
SP
Shree Pandya
ROCHESTER, NY
Specialist
2 Duchenne and Becker muscular dystrophy publications
CO
Carlos Ortez
Specialist
2 Duchenne and Becker muscular dystrophy publications

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Duchenne and Becker muscular dystrophy.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Duchenne and Becker muscular dystrophyForum →

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Latest news about Duchenne and Becker muscular dystrophy

No recent news articles for Duchenne and Becker muscular dystrophy.

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Duchenne and Becker muscular dystrophy

What is Duchenne and Becker muscular dystrophy?

Duchenne and Becker Muscular Dystrophy is treated with 3 medications in our database, including Deflazacort, DEFLAZACORT, Vyondys 53, JAYTHARI Oral Suspension. 2 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by PTC Therapeutics, Sarepta, Amneal. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Duchenne and Becker Muscular Dystrophy treatment below.

How is Duchenne and Becker muscular dystrophy inherited?

Duchenne and Becker muscular dystrophy follows a x-linked recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Duchenne and Becker muscular dystrophy typically begin?

Typical onset of Duchenne and Becker muscular dystrophy is childhood. Age of onset can vary across affected individuals.

Which specialists treat Duchenne and Becker muscular dystrophy?

10 specialists and care centers treating Duchenne and Becker muscular dystrophy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Duchenne and Becker muscular dystrophy?

3 FDA-approved treatments are currently tracked on UniteRare for Duchenne and Becker muscular dystrophy. See the treatments and support programs sections for copay assistance, eligibility, and contact details.