Overview
Pompe disease is treated with 1 medication in our database, including Nexviazyme. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Sanofi. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Pompe disease treatment below.
Autosomal recessive
Passed on when both parents carry the same gene change; often skips generations
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
1 eventData sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
3 availableLumizyme
1 INDICATIONS AND USAGE LUMIZYME ® is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase [GAA] deficiency). LUMIZYME ® is a hydrolytic lysoso…
1 INDICATIONS AND USAGE LUMIZYME ® is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase [GAA] deficiency). LUMIZYME ® is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency). ( 1 )
Nexviazyme
1 INDICATIONS AND USAGE NEXVIAZYME is indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency). NEXVIAZYME is a…
1 INDICATIONS AND USAGE NEXVIAZYME is indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency). NEXVIAZYME is a hydrolytic lysosomal glycogen-specific enzyme indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency). ( 1 )
POMBILITI ATGA
1 INDICATIONS AND USAGE POMBILITI is indicated, in combination with Opfolda, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weigh…
1 INDICATIONS AND USAGE POMBILITI is indicated, in combination with Opfolda, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). POMBILITI is a hydrolytic lysosomal glycogen-specific enzyme indicated, in combination with Opfolda, an enzyme stabilizer, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). ( 1 )
Rare Disease Specialist
Rare Disease Specialist
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Financial Resources
5 resourcesNORD Patient Assistance — Pompe disease
NORD Patient Assistance
Pompe disease
The Assistance Fund — Pompe disease
The Assistance Fund
Pompe disease
HealthWell Foundation — Pompe disease
HealthWell Foundation
Pompe disease
6 travel grants are also available for Pompe disease patients — see Travel Grants below ↓
Travel Grants
6 grantsPompe disease medication copay assistance - PAN Foundation
PAN Foundation
Applicants must be receiving treatment for Pompe disease in the United States and have health insurance that covers their qualifying medication. Eligible patients must also have a household income at or below 500% of the Federal Poverty Level.
PAN: Pompe Disease Financial Assistance
PAN: Pompe Disease (Premium Assistance)
Patients must have a diagnosis of Pompe Disease and reside in the United States or Puerto Rico. Eligibility requires enrollment in Medicare, Medicaid, or commercial insurance for this needs-based premium assistance fund.
The Assistance Fund: Pompe Disease
Community
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1 articlesCaregiver 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 Pompe disease
What is Pompe disease?
Pompe disease is treated with 1 medication in our database, including Nexviazyme. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Sanofi. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Pompe disease treatment below.
How is Pompe disease inherited?
Pompe disease follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Are there clinical trials for Pompe disease?
Yes — 1 recruiting clinical trial is currently listed for Pompe disease on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Pompe disease?
17 specialists and care centers treating Pompe disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Pompe disease?
1 FDA-approved treatment and 6 patient support programs are currently tracked on UniteRare for Pompe disease. See the treatments and support programs sections for copay assistance, eligibility, and contact details.