Patient Advocacy Groups h1 >
![AMDA logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-AMDA.jpg)
Acid Maltase Deficiency Association
Funds research and promotes public awareness of acid maltase deficiency, also known as Pompe’s disease.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-ACF.jpg)
Addi & Cassi Fund (For Niemann-Pick disease type C)
Advocates for individuals with Niemann-Pick type C and their families.
![APMRF logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-APMRF.jpg)
Ara Parseghian Medical Research Foundation
Aims to find a treatment or cure for Niemann-Pick type C (NPC) disease.
![AGSD logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-AGSDUS.jpg)
Association for Glycogen Storage Disease
Supports individuals with glycogen storage disease (GSD) and their families through information, awareness, and support of research.
![APA logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-AUS-POMPE.jpg)
Australian Pompe's Association, Inc.
Supports the needs of Australian Pompe's disease patients, their families, and caregivers.
![BDSRA logo](https://static.rarediseasesnetwork.org/logos-pags/LDN_PAGs_BDSRA-Name%2BTag_2022.png)
Batten Disease Support and Research Association
Determined to provide unwavering support, fund progressive research, and be a source of steadfast advocacy for all ceroid lipofuscinosis, neuronal (CLN) types.
![Ben's Dream logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-BensDream.jpg)
Ben's Dream Sanfilippo Research Foundation
Promotes awareness and raises funds to support research aimed at finding a cure for all types of Sanfilippo syndrome.
![CAP logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-POMPE-Canada.jpg)
Canadian Association of Pompe
Helps Pompe patients and their families in Canada through education, support, and community.
![CFA logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-FABRYCANADA.jpg)
Canadian Fabry Association
Encourages and supports research into the causes, treatment, and management of Fabry disease.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-MPSSOCIETY-CA.jpg)
Canadian MPS Society
Offers support and education programs for those managing the diagnosis and progression of mucopolysaccharide (MPS) and related diseases.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-GRAY-FOUNDATION.jpg)
Charlotte and Gwenyth Gray Foundation
Works to improve and save the lives of all children impacted by Batten disease and other neurodegenerative disorders.
![CGRF logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-childrensgaucher.jpg)
Children's Gaucher Disease Research Fund
Unites families and raises funds to support research to find a cure for type 2 and type 3 Gaucher disease.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-CHLOES-FIGHT.jpg)
Chloe's Fight Rare Disease Foundation
Raises awareness for all rare diseases and funds research related to leukodystrophies.
![Cure GM1 logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-CUREGM1.jpg)
Cure GM1 Foundation
Funds research to benefit all individuals affected by GM1 gangliosidosis.
![Cure ML logo](https://static.rarediseasesnetwork.org/logos-pags/CureMucolipidosisLogo.jpg)
Cure Mucolipidosis
Aims to develop a pipeline of therapies for mucolipidosis alpha/beta, mucolipidosis gamma, and sialidosis.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-CURESFF.jpg)
Cure Sanfilippo Foundation
Accelerates research to find the first-ever FDA-approved treatment or cure for Sanfilippo syndrome.
![CTSF logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-CURETAY-SACHS.jpg)
Cure Tay-Sachs Foundation
Funds the ongoing research needed to find treatments and a cure for Tay-Sachs disease while promoting carrier testing and awareness initiatives.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-cystinosisfoundation.jpg)
Cystinosis Foundation
Educates and supports patients, families, and medical professionals for cystinosis.
![CRF logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-cystinosisresearch.jpg)
Cystinosis Research Foundation
Supports bench, clinical, and translational research to find better treatments and a cure for cystinosis.
![CRN logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-cystinosisresearchnetwork.jpg)
Cystinosis Research Network
Supports and advocates for research, provides family assistance, and educates the public and medical communities about cystinosis.
![logo](https://static.rarediseasesnetwork.org/logos-pags/pag-danas-angels.jpg)
Dana’s Angels Research Trust
Funds medical research, medical education, or medical or hospital care for the treatment or cure of Niemann-Pick Type C disease (NPC) or other similar genetic diseases.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-FABRY-AU.jpg)
Fabry Australia, Inc.
Unites and supports the Australian Fabry community.
![FSIG logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-FABRY.jpg)
Fabry Support & Information Group
Supports the needs of the Fabry disease community by providing information, education, and advocacy.
![GLSDSA logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-GAUCHER.jpg)
Gaucher & LSD Society of South Africa
Aims to improve the lives of all lysosomal storage disease patients in South Africa.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-hideandseek.jpg)
Hide and Seek Foundation for LDN Research
Works to find a cure for the lysosomal disease Niemann-Pick type C (NPC).
![Hunter's Hope logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-HuntersHope.jpg)
Hunter's Hope Foundation
Supports and encourages those affected by Krabbe disease and related leukodystrophies.
![ISMRD logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-ISMRD.jpg)
International Society for Mannosidosis and Related Diseases
Advocates for families and patients affected by glycoprotein diseases.
![JJB logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-JonahsJustBegun.jpg)
Jonah's Just Begun - Foundation to Cure Sanfilippo, Inc
Raises and distributes funds to academic research groups focused on finding treatments for Sanfilippo syndrome, mucopolysaccharidosis (MPS) III.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-KRABBECONNECT.jpg)
KrabbeConnect
Provides a platform to amplify the voice of patients, aiding researchers and drug developers in accelerating research for better treatments for Krabbe disease.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-LALDAWARE.jpg)
LAL-D Aware
Supports children and adults with lysosomal acid lipase (LAL) deficiency, cholesteryl ester storage disease (CESD), and Wolman disease.
![LDNZ logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-LDNZ.jpg)
Lysosomal Diseases New Zealand
Provides information on rare lysosomal diseases and support for affected families in New Zealand.
![LSDSS logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-LSDSS.jpg)
Lysosomal Storage Disorders Support Society
Works to raise awareness and offer education and support on lysosomal storage disorders in India.
![logo](https://static.rarediseasesnetwork.org/logos-pags/GLIA-CTN_PAGs_MLD-Foundation_543x196.png)
MLD Foundation
Provides compassion for families, increases awareness, influences and funds research, and promotes education for metachromatic leukodystrophy.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-MLD-SUPPORT-UK.jpg)
MLD Support Association UK
Aims to bring hope to families in the fight to eradicate metachromatic leukodystrophy (MLD) by providing support and information on research and treatments.
![logo](https://static.rarediseasesnetwork.org/logos-pags/GLIA-CTN_PAGs_Saving-Dylan_543x196.png)
MSD Action Foundation
Promotes and supports research leading to positive clinical outcomes and quality of life for patients affected by multiple sulfatase deficiency.
![ML4 Foundation logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-ML4.jpg)
Mucolipidosis IV Foundation
Funds, promotes, and supports medical research dedicated to developing treatments and a cure for ML4.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-NathansBattle.jpg)
Nathan's Battle Foundation
Utilizes entrepreneurial spirit and techniques to seek out and develop promising therapeutic approaches for late infantile Batten disease.
![NFDF logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-NFDF.jpg)
National Fabry Disease Foundation
Assists with disease education, diagnosis, support, research, and advovcacy for the Fabry disease community.
![NGF logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-NGF.jpg)
National Gaucher Foundation
Serves U.S. patients with Gaucher disease and their families through financial support, education, patient services, and collaboration with medical professionals.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-NMPSS.jpg)
National Mucopolysaccharidoses (MPS) Society
Provides support for families and fosters research for a cure for mucopolysaccharide (MPS) disease.
![NNPDF logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-NNPDF.jpg)
National Niemann-Pick Disease Foundation Inc.
Supports and empowers patients and families affected by Niemann-Pick disease through education, collaboration, and research.
![NTSAD logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-NTSAD.jpg)
National Tay-Sachs & Allied Diseases Association, Inc.
Aims to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases by driving research, forging collaboration, and fostering community.
![logo](https://static.rarediseasesnetwork.org/logos-pags/NewHope.jpg)
New Hope Research Foundation
Aims to provide hope to families and find a genetic cure for GM2 gangliosidosis (Tay-Sachs Disease) and other lysosomal storage diseases of the central nervous system.
![NPUK logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-NPUK.jpg)
Niemann-Pick UK
Aims to make a positive difference in the lives of those affected by Niemann-Pick disease and their families, from diagnosis and beyond.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-PROJECT-ALIVE.jpg)
Project Alive (for Hunter syndrome)
Serves as a voice for children and adults with Hunter syndrome (mucopolysaccharidosis or MPS II), working to find a cure through research and advocacy.
![RQMO logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-RQMO.jpg)
Quebec Coalition of Orphan Diseases
Offrir un service d'information et de soutien pour les malades atteints d'une maladie rare et orpheline, leurs familles et pour les professionnels de la santé.
![RDNZ logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-RDNZ.jpg)
Rare Disorders NZ
Offers a starting point for patients and families affected by rare disorders, helping families, patients, and healthcare providers find essential information and support groups.
![logo](https://static.rarediseasesnetwork.org/logos-pags/RareXLogo.jpg)
RARE-X
Focuses on supporting the acceleration and development of life-altering treatments and future cures for patients impacted by a rare disease.
![RFRF logo](https://static.rarediseasesnetwork.org/logos-pags/ldn-pag-rosenau-family-foundation.jpg)
Rosenau Family Research Foundation
Works to promote the expansion of newborn screening and to further education, awareness, and research of Krabbe disease and cystic fibrosis.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-SALLA.jpg)
Salla Treatment and Research Foundation
Supports Salla disease treatments, research, education, awareness, and family networks.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-SANFILLIPPO-AU.jpg)
Sanfilippo Children's Foundation
Drives research for a world without Sanfilippo syndrome by funding research, providing information, raising awareness, advocating, and improving diagnosis.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-TaylorsTale.jpg)
Taylor's Tale
Works to build a better future for Americans with rare diseases by advancing breakthrough treatments for Batten disease and advocating for life-changing public policies.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-IsaacFoundation.jpg)
The Isaac Foundation
Funds research projects that aim to find a cure for mucopolysaccharidosis (MPS); advocates and provides support for families of individuals suffering from MPS.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-GAUCHER-CA.jpg)
The National Gaucher Foundation of Canada
Aims to improve the health and wellbeing of Canadian Gaucher patients through education, advocacy, and support of research.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-QUINN-MADELEINE.jpg)
The Quinn Madeleine Foundation
Aids in the prevention of Niemann-Pick disease type A (also known as ASMD), as well as supports research on treatments and a cure.
![logo](https://static.rarediseasesnetwork.org/logos-pags/ldn-ryan-foundation.jpg)
The Ryan Foundation
Raises awareness and funding for orphan disease research, with a focus on mucopolysaccharidos type I (MPS I) and related diseases.
![logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-SCRF.jpg)
The Sanfilippo Children's Research Foundation
Funds medical research into Sanfilippo syndrome and its related neurogenetic diseases.
![MPS Society logo](https://static.rarediseasesnetwork.org/logos-pags/Logo-PAG-MPSSOCIETY-UK.jpg)
The Society for Mucopolysaccharide Diseases (UK)
Provides support across the United Kingdom to families affected by one of 25 rare, life-limiting genetic conditions.
![logo](https://static.rarediseasesnetwork.org/logos-pags/GLIA-CTN_PAGs_United-Leukodystrophy-Foundation_543x196.png)
United Leukodystrophy Foundation (ULF)
ULF is a non-profit, voluntary health organization dedicated to funding cutting-edge research and to providing patients and their families with disease information and medical referrals.
![Cure MSD logo](https://static.rarediseasesnetwork.org/logos-pags/GLIA-CTN_PAGs_United-MSD-Foundation_543x196.png)
United MSD Foundation
Aspires for all children with multiple sulfatase deficiency (MSD) to lead a healthy life without limitations.
![logo](https://static.rarediseasesnetwork.org/logos-pags/united-pompe-foundation.jpg)
United Pompe Foundation
Assists patients and/or their families with medical costs and other expenses related to Pome Disease.