amda pompe

What amda pompe does

AMDA Pompe (Acid Maltase Deficiency Association) is a 501(c)(3) nonprofit patient advocacy organization founded in 2011 by the House family and headquartered in San Antonio, Texas. The organization serves individuals and families affected by Pompe disease, a rare genetic lysosomal storage disorder, through educational programming, peer support networks, patient registries, advocacy campaigns, and research funding. AMDA's core "products" are non-commercial services—webinars, newsletters, the TWYPP peer support program, biannual conferences, and Rare Disease Week advocacy initiatives—rather than technology platforms or licensed intellectual property. The organization does not develop software or commercial technology; its infrastructure consists of community programming, content distribution, and convening activities.

The organization's business model is donation-based rather than commercial. Revenue derives from individual contributions, foundation grants, and pharmaceutical partnership support, with documented collaborators including Sanofi Genzyme, Astellas Gene Therapies, Spark Therapeutics, and Ask Bio on the industry side, and Erasmus MC and the International Pompe Association on the research and international coordination side. AMDA plays a convening role across patient, clinical, academic, and commercial Pompe disease stakeholders, and is positioned within the U.S. rare disease policy ecosystem through FDA advisory committee engagement. The organization experienced a significant leadership inflection point with the death of founder Tiffany L. House on May 25, 2025, which introduces near-term continuity risk for donor relationships and strategic direction while leaving programmatic activities and partnerships intact as of the latest available information.

Name

amda pompe

Legal name

Acid Maltase Deficiency Association

Website

https://amda-pompe.org

Company type

Private

Founded year

2011

Operating status

Operating

Short description

AMDA Pompe is a 501(c)(3) nonprofit patient advocacy organization founded in 2011 and headquartered in San Antonio, Texas, that supports individuals and families affected by Pompe disease through education, peer support, research funding, and rare disease policy advocacy in partnership with international research institutions and pharmaceutical sponsors.

Ownership category

akta.pro rank

Headquarters

HQ city

San Antonio

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

Personnel, Operations, Others, Marketing or Sales, Technology or R&D

Distribution channels1 record

Marketing channels8 records

Core offering

AMDA Pompe advances research, improves care, and ensures early diagnosis for individuals and families affected by Pompe disease. It delivers educational webinars, peer support sessions, a patient registry, biannual conferences, the Helen Walker Research Grant for scientific research, Emergency Medical Information & Alert Cards, and Continuing Medical Education resources. The organization advocates for the Pompe community with regulators, policymakers, and industry partners globally.

Product overview

AMDA (Acid Maltase Deficiency Association) is a patient advocacy nonprofit organization that does not offer commercial products or services. It provides support programs for the Pompe disease community, including educational webinars, patient registry services, research funding through the Helen Walker Research Grant, conferences, newsletters, and resources for patients and healthcare professionals. The organization facilitates collaboration between patients, families, researchers, industry professionals, and policymakers to advance Pompe disease awareness, early diagnosis, and treatment.

Differentiator

Problem solved

Functional benefit

Products and services

• Educational Webinar Program Recurring webinar series featuring expert speakers on Pompe disease diagnosis, enzyme replacement therapy, gene therapy, newborn screening, patient registries, mental health, and daily living management; intended for Pompe patients, families, and healthcare professionals.
• Patient Registry Online patient registry enabling Pompe disease patients to enroll and contribute data that supports research and patient-reported outcomes; aligned with global efforts such as the IPA/Erasmus MC Pompe Survey.
• Helen Walker Research Grant Research funding program administered by AMDA's Scientific Board to advance Pompe disease scientific research.
• Biannual Patient and Scientific Conferences Conferences held twice per year that bring together Pompe disease patients, families, researchers, and healthcare professionals for scientific exchange and community building.
• Talking With Your Pompe Peeps (TWYPP) Peer Support Sessions Peer support discussion sessions moderated by community members with lived Pompe disease experience, covering topics such as college life, service dogs, home infusions, diet, and holiday planning.
• Emergency Medical Information & Alert Card Emergency medical information resource and alert card for Pompe disease patients to use during medical emergencies and care transitions.
• Continuing Medical Education (CME) Program Continuing medical education resources and programming provided for healthcare professionals focused on Pompe disease management.
• AMDA Newsletter Email newsletter providing announcements of new webinars, news, events, and resources to the Pompe disease community.

Segments2 records

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Partnerships

Ten partnerships are on record, tiered core, minor and moderate.

• International Pompe Association (IPA)coreStrategic or Co-development PartnerAMDA works closely with the International Pompe Association, an international Pompe patient support organization. AMDA President Tiffany House served as Chairman/Board Member of IPA. The IPA/Erasmus MC Pompe Survey (launched 2002) collects patient-reported outcomes from Pompe patients worldwide to track disease impacts and treatment effects.
• Erasmus MC University Medical Center, Rotterdam, NetherlandscoreStrategic or Co-development PartnerCollaboration on the IPA/Erasmus MC Pompe Survey since 2002, which collects information on how Pompe disease impacts patients' lives and how these impacts may change with treatment. The center's researchers present at AMDA webinars.
• Sanofi GenzymeminorGTM or Marketing PartnerSanofi has collaborated with AMDA on webinars and the Sanofi Genzyme Pompe Registry. AMDA held an AMDA Zoom Webinar with Sanofi in April 2022 on the Pompe Registry.
• Astellas Gene TherapiesminorGTM or Marketing PartnerAstellas Gene Therapies presented a webinar on evaluating gene therapies for Pompe disease, including clinical trial goals and assessments.
• Spark TherapeuticsminorGTM or Marketing PartnerSpark Therapeutics collaborated on webinars about gene therapy research for Late-Onset Pompe Disease. Multiple Spark-sponsored webinars have been hosted on the AMDA platform.
• Ask BiominorGTM or Marketing PartnerAsk Bio presented a webinar on Development of Gene Therapy for Pompe Disease in November 2023.
• Boston Children's HospitalminorStrategic or Co-development PartnerBoston Children's Hospital researchers presented a webinar on defining central nervous system abnormalities in Pompe disease patients in October 2023.
• Duke University Medical CentercoreStrategic or Co-development PartnerDr. Priya Kishnani from Duke University is a key opinion leader in Pompe disease. Duke played an integral role in the clinical development of alglucosidase alfa and avalglucosidase alfa. Duke researchers present regularly at AMDA webinars.
• Lysosomal Storage Disorders (LSD) Advocacy CoalitionmoderateGTM or Marketing PartnerAMDA participates in monthly meetings to work together on state and federal issues including Medicare Home Infusion, medical research, and Newborn Screening advocacy.
• Lysosomal Disease Network (LDN) - NIH Rare Diseases Clinical Research NetworkmoderateStrategic or Co-development PartnerTiffany House served as an Active/Voting Member of the LDN External Advisory Committee, evaluating pilot project proposals and fellowship applications with focus on MPS, Pompe, and Fabry diseases.

Scale indicators2 records

Recent moves5 records

Expansion highlights5 records

Emerging players

• Acid Lipase Disorders Association (ALDA): EMERGING_PLAYER: peer nonprofit advocacy organization for another lysosomal storage disorder (Wolman disease / Cholesteryl Ester Storage Disease). Operates a similar patient education and research-funding model for a comparably rare LSD population.
• Fabry Support & Information Group: EMERGING_PLAYER: peer patient advocacy organization for Fabry disease, another lysosomal storage disorder with enzyme replacement and gene therapy pipelines. Operates a similar education/registry/research-funding model for the Fabry community.
• National Niemann-Pick Disease Foundation: EMERGING_PLAYER: peer nonprofit patient advocacy and research-funding organization for Niemann-Pick disease, another lysosomal storage disorder. Similar size, structure, and mission to AMDA.
• Cure SMA (Spinal Muscular Atrophy): EMERGING_PLAYER: peer disease-specific patient advocacy and research-funding organization for SMA, with a similar model (research grants, patient registry, family support, gene therapy/ERT education) for another rare neuromuscular disease.
• United Leukodystrophy Foundation: EMERGING_PLAYER: peer rare-disease patient advocacy organization supporting families affected by leukodystrophies, with a comparable education and research-funding operating model targeted at a similarly small ultra-rare population.

Others

• National Organization for Rare Disorders (NORD): OTHERS / BROAD_INCUMBENT: umbrella advocacy organization representing ~300 rare disease patient communities including Pompe. AMDA participates alongside NORD in newborn screening and policy coalitions; NORD is complementary rather than directly competitive.

Direct peers

• International Pompe Association (IPA): Direct peer: international umbrella organization for Pompe disease patient associations. AMDA works closely with IPA through its former President Tiffany House (IPA Chairman/Board Member) and co-runs the IPA/Erasmus MC Pompe Survey with Erasmus MC.

Broad incumbents

• Muscular Dystrophy Association (MDA): BROAD_INCUMBENT: large established patient advocacy and research-funding organization covering neuromuscular diseases. Pompe disease overlaps with MDA's mission; AMDA is a more disease-specific niche alternative within that broader neuromuscular advocacy category.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks5 records

Key highlights5 records

Customer concentration

• YouTubeyoutube.com/channel/UCK3be10hagtoHXVlIklcv8A
• Facebookfacebook.com/AcidMaltaseDeficiencyAssociation
• Instagraminstagram.com/amda_association
• LinkedInlinkedin.com/company/acid-maltase-deficiency-association-inc

Revenue estimate

Valuation estimate

Number of profiles

Profiles2 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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