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Patient Advocacy Groups

AMDA logo

Acid Maltase Deficiency Association

Funds research and promotes public awareness of acid maltase deficiency, also known as Pompe’s disease.

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Addi & Cassi Fund (For Niemann-Pick disease type C)

Advocates for individuals with Niemann-Pick type C and their families.

ALD Foundation logo

Adrenoleukodystrophy Foundation

Advocates for patients with adrenoleukodystrophy (ALD) and adrenomyeloneuropathy (AMN).

APMRF logo

Ara Parseghian Medical Research Foundation

Aims to find a treatment or cure for Niemann-Pick type C (NPC) disease.

AGSD logo

Association for Glycogen Storage Disease

Supports individuals with glycogen storage disease (GSD) and their families through information, awareness, and support of research.

APA logo

Australian Pompe's Association, Inc.

Supports the needs of Australian Pompe's disease patients, their families, and caregivers.

BDSRA logo

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

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

Canadian Association of Pompe

Helps Pompe patients and their families in Canada through education, support, and community.

CFA logo

Canadian Fabry Association

Encourages and supports research into the causes, treatment, and management of Fabry disease.

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Canadian MPS Society

Offers support and education programs for those managing the diagnosis and progression of mucopolysaccharide (MPS) and related diseases.

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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

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.

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Chloe's Fight Rare Disease Foundation

Raises awareness for all rare diseases and funds research related to leukodystrophies.

Cure GM1 logo

Cure GM1 Foundation

Funds research to benefit all individuals affected by GM1 gangliosidosis.

Cure ML logo

Cure Mucolipidosis

Aims to develop a pipeline of therapies for mucolipidosis alpha/beta, mucolipidosis gamma, and sialidosis.

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Cure Sanfilippo Foundation

Accelerates research to find the first-ever FDA-approved treatment or cure for Sanfilippo syndrome.

CTSF logo

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.

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Cystinosis Foundation

Educates and supports patients, families, and medical professionals for cystinosis.

CRF logo

Cystinosis Research Foundation

Supports bench, clinical, and translational research to find better treatments and a cure for cystinosis.

CRN logo

Cystinosis Research Network

Supports and advocates for research, provides family assistance, and educates the public and medical communities about cystinosis.

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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.

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Fabry Australia, Inc.

Unites and supports the Australian Fabry community.

FSIG logo

Fabry Support & Information Group

Supports the needs of the Fabry disease community by providing information, education, and advocacy.

GLSDSA logo

Gaucher & LSD Society of South Africa

Aims to improve the lives of all lysosomal storage disease patients in South Africa.

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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

Hunter's Hope Foundation

Supports and encourages those affected by Krabbe disease and related leukodystrophies.

ISMRD logo

International Society for Mannosidosis and Related Diseases

Advocates for families and patients affected by glycoprotein diseases.

JJB logo

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.

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KrabbeConnect

Provides a platform to amplify the voice of patients, aiding researchers and drug developers in accelerating research for better treatments for Krabbe disease.

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LAL-D Aware

Supports children and adults with lysosomal acid lipase (LAL) deficiency, cholesteryl ester storage disease (CESD), and Wolman disease.

LDNZ logo

Lysosomal Diseases New Zealand

Provides information on rare lysosomal diseases and support for affected families in New Zealand.

LSDSS logo

Lysosomal Storage Disorders Support Society

Works to raise awareness and offer education and support on lysosomal storage disorders in India.

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MLD Foundation

Provides compassion for families, increases awareness, influences and funds research, and promotes education for metachromatic leukodystrophy.

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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.

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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

Mucolipidosis IV Foundation

Funds, promotes, and supports medical research dedicated to developing treatments and a cure for ML4.

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Nathan's Battle Foundation

Utilizes entrepreneurial spirit and techniques to seek out and develop promising therapeutic approaches for late infantile Batten disease.

NFDF logo

National Fabry Disease Foundation

Assists with disease education, diagnosis, support, research, and advovcacy for the Fabry disease community.

NGF logo

National Gaucher Foundation

Serves U.S. patients with Gaucher disease and their families through financial support, education, patient services, and collaboration with medical professionals.

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National Mucopolysaccharidoses (MPS) Society

Provides support for families and fosters research for a cure for mucopolysaccharide (MPS) disease.

NNPDF logo

National Niemann-Pick Disease Foundation Inc.

Supports and empowers patients and families affected by Niemann-Pick disease through education, collaboration, and research.

NTSAD logo

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.

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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

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.

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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

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

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.

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RARE-X

Focuses on supporting the acceleration and development of life-altering treatments and future cures for patients impacted by a rare disease.

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Salla Treatment and Research Foundation

Supports Salla disease treatments, research, education, awareness, and family networks.

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Sanfilippo Children's Foundation

Drives research for a world without Sanfilippo syndrome by funding research, providing information, raising awareness, advocating, and improving diagnosis.

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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.

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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.

TLOAF logo

The Legacy of Angels Foundation

Works to promote the expansion of newborn screening and to further education, awareness, and research of Krabbe disease and cystic fibrosis.

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The National Gaucher Foundation of Canada

Aims to improve the health and wellbeing of Canadian Gaucher patients through education, advocacy, and support of research.

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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.

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The Ryan Foundation

Raises awareness and funding for orphan disease research, with a focus on mucopolysaccharidos type I (MPS I) and related diseases.

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The Sanfilippo Children's Research Foundation

Funds medical research into Sanfilippo syndrome and its related neurogenetic diseases.

MPS Society logo

The Society for Mucopolysaccharide Diseases (UK)

Provides support across the United Kingdom to families affected by one of 25 rare, life-limiting genetic conditions.

Cure MSD logo

United MSD Foundation

Aspires for all children with multiple sulfatase deficiency (MSD) to lead a healthy life without limitations.

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United Pompe Foundation

Assists patients and/or their families with medical costs and other expenses related to Pome Disease.