We Involve Advocacy Organizations in the Conversation.
The following organizations provide educational resources and support research to help individuals affected lysosomal storage disorders (LSDs).
Below are examples of the communities we serve:
The Fabry Support & Information Group (FSIG) is dedicated to raising awareness of Fabry disease and its symptoms, advocating for community needs, and leading the fight for the most effective treatment possible and eventually a cure. FSIG provides a link for the Fabry community to information, each other, the medical community, researchers, the pharmaceutical industry, and regulatory authorities. FSIG strives to support affected families and individuals lead fuller lives.
The National Fabry Disease Foundation (NFDF) is dedicated to supporting the Fabry disease community. Its five primary functions are assisting with Fabry disease education, facilitating Fabry disease identification to improve Fabry disease recognition and diagnosis, providing various forms of assistance to individuals with Fabry disease, supporting Fabry disease research, and promoting advocacy for Fabry disease issues.
The Fabry International Network (FIN) is dedicated to providing information, communications, actions, and decisions to patients all over the world in a neutral and independent manner. The FIN seeks to see the implementation of a global Fabry Expert Meeting that allows a platform where all relevant aspects and information of Fabry disease, current or future drugs and therapies can be shared in a non-biased.
The National Gaucher Foundation (NGF) is dedicated to serving U.S. patients with Gaucher disease and their families. Through financial support, educational programming, patient services, and collaboration with medical professionals, the NGF empowers Gaucher patients to live a better today.
The International Gaucher Alliance (IGA) is a patient-led international organization that has become the ‘go to’ global voice for over 85% of the Gaucher community and has built its reputation through listening to and delivering outcomes that have impacted on patients and their caregivers’ lives.
The Gaucher Community Alliance (GCA) is a patient-led organization, and its mission is to support patients with Gaucher disease and their families through peer-to-peer support and education, advocacy, patient and family resources, and networking. Increased collaboration and emotional support among patients living with all types of Gaucher disease may alleviate some of the burden associated with this rare disorder.
Genetic Alliance (GA) is dedicated to transforming health through engages individuals, families and communities. It partners with individuals and communities to transform health systems to respond to what people most need. Th GA convenes powerful networks, deliver actionable information, build intuitive tools, and drive policy decisions.
The International Advocate for Glycoprotein Storage Diseases (ISMRD) is the leading advocate for families worldwide affected by a Glycoprotein Storage Disease. Through partnerships built with medicine, science, and industry, it seeks to detect and cure these diseases, and to provide a global network of support and information.
The Canadian MPS Society is dedicated to easing the burden of an MPS diagnosis by directing families to important sources of information, research, and medical help, and by connecting them with other families experiencing similar situations to expand their network of support.
The Cure Sanfilippo Foundation is committed to advocating for and funding research that could give children with Sanfilippo syndrome a chance at a better life by finding a treatment or cure for the disease. It works diligently to ensure research funded by the Foundation is well rounded and includes the study of all disease subtypes and stages.
The MPS Society UK is dedicated to transforming lives through support, research, and awareness. The Society professional support to individuals and families affected by MPS, Fabry or a related disease in the UK.
The National MPS Society is dedicated to finding a cure, support, and advocate for mucopolysaccharidosis (MPS) and mucolipidosis (ML). Its mission is to serve individuals, families, and friends affected by MPS to support research, to support families, and to increase public and professional awareness.
Team Sanfilippo Foundation is a medical research foundation founded in 2008 by parents of children with Sanfilippo Syndrome. Its mission is to fund potential therapies that can be in clinical trials soon. It supports biotechnology and pharmaceutical companies and research centers with potential therapies that are underfunded and aid with connecting families to companies that need information for ongoing clinical work.
The International Pompe Association (IPA) is a federation of Pompe disease patient’s groups world-wide. It seeks to coordinate activities and share experience and knowledge between different groups. Its objectives are to stimulate research into the causes, treatment, and prevention of Pompe disease.
The Association for Glycogen Storage Disease is dedicated to protecting and promoting the best interests of all persons and families affected by Glycogen Storage Disease [GSD]; to promote the establishment, improvement, and management of facilities for the treatment, study, education, shelter, recreation, recuperation and other general benefit to GSD-affected persons; to act as a vehicle of communication on GSD-related matters to patients, families, professionals, and the public; and to act as a focus for scientific, educational, and charitable activities related to the disease.
The Metabolic Support UK is committed to becoming the world’s leading advocate and provider of bespoke support to families; signpost to the best experts; raise awareness among the medical, healthcare, social care and teaching professions; and fund research for treatments and ultimately cures.
The United Pompe Foundation is dedicated to assisting patients and/or their families with medical costs and other expenses that these patients and families face and may not be able to cover, or fully cover, through their insurance.
The Acid Maltase Deficiency Association, Inc. (AMDA) is dedicated toward the advancement of research in acid maltase deficiency (also known as Pompe disease). The association publishes brochures and informational literature; sponsors an annual conference for the scientific community to discuss crucial issues related to Pompe disease; and maintains a patient/family registry.
Global Genes provides hope for the more than 400 million people affected by rare disease around the globe. They fulfill their mission by helping patients find and build communities, gain access to information and resources, connect to researchers, clinicians, industry, government, and other stakeholders, share data and experiences, stand up, stand out, and become effective advocates on their behalf.
The EveryLife Foundation for Rare Diseases is a nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments, and cures.
The National Organization for Rare Disorders (NORD) is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 280 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.
EURORDIS (Rare Diseases Europe) is a non-governmental patient-driven alliance of patient organizations representing 962 rare disease patient organizations in 73 countries. EURORDIS is the voice of 30 million people affected by rare diseases throughout Europe.