Rare Disease Report

Appendix B: Patient Advocacy Groups


Martin Mueller IV Achalasia Awareness Foundation
Acid Maltese Deficiency

Acid Maltase Deficiency Association (AMDA)

Acromegaly Community, Inc.
Adult Polyglucosan Body Disease

Adult Polyglucosan Body Disease Research Foundation
APBD Research Foundation

AKU Society of North America
Adrenal Diseases

National Adrenal Diseases Foundation
Alagille Syndrome

Alagille Syndrome Alliance

National Organization for Albinism and Hypopigmentation, NOAH
Alopecia Areata

National Alopecia Areata Foundation
Alpha 1

Alpha 1 Association
Alport Syndrome

Alport Syndrome Foundation
Alternating Hemiplegia of Childhood

Alternating Hemiplegia of Childhood Foundation
Cure AHC
Amyloid Lateral Sclerosis

ALS Association-Greater Philadelphia Chapter
Les Turner ALS Foundation, Ltd.
ALS Association

Amyloidosis Support Groups, Inc
Aplastic Anemia

Aplastic Anemia & MDS International Foundation, Inc.
Julia’s Wings Foundation, Inc

A-T Children's Project
National Ataxia Foundation
Autoimmune Diseases

American Autoimmune Related Diseases Association, Inc.
Autoinflammatory Diseases

NOMID Alliance
Basal Cell Carcinoma Nevus Syndrome

Basal Cell Carcinoma Nevus Syndrome Life Support Network
Batten Disease

Batten Disease Support and Research Association 
Behcet’s Disease

American Behcet's Disease Association 
Benign Essential Blepharospasm

Benign Essential Blepharospasm Research Foundation, Inc. 
Bile Acid Deficiency

Council for Bile Acid Deficiency Diseases 
Blindness (rare forms)

Foundation Fighting Blindness 
Brain Tumor

American Brain Tumor Association
National Brain Tumor Society

CADASIL Association, Inc. 
Cancer (pediatric)

Caleb’s Crusade
Children's Tumor Foundation
Smashing Walnuts Foundation
Cancer (rare forms)

Rare Cancer Research Foundation
Carcinoid Cancer

Caring for Carcinoid Foundation 
Cardiofaciocutaneous Syndrome

Cardio-Facio-Cutaneous International
Cardiomyopathy (pediatric)

Children's Cardiomyopathy Foundation

International Foundation for CDKL5 Research
Charlot-Marie-Tooth Disease

Charcot-Marie-Tooth Association 

Cholangiocarcinoma Foundation
Chronic Fatigue Syndrome

Wisconsin ME/CFS Association

Clostridium Septicum
Gut Check Foundation
Craniofacial Disorders

American Cleft Palate-Craniofacial Association
Children's Craniofacial Association
Cleft Palate Foundation
Chromosome Disorders

Chromosome 18 Registry & Research Society
Chromosome Disorder Outreach, Inc.
Chronic Granulomatous Disease

Chronic Granulomatous Disease Association 
Cicatricial Alopecia

Cicatricial Alopecia Research Foundation
CLOVES Syndrome

CLOVES Syndrome Community 
Coats Disease

Jack McGovern Coats Disease Foundation 
Congenital Central Hypoventilation Syndrome

CCHS Family Network
Congenital Diaphragmatic Hernia

Breath of Hope, Inc.
Congenital Hyperinsulinism

Congenital Hyperinsulinism International
Cornelia de Lange Syndrome

Cornelia de Lange Syndrome Foundation, Inc. 
Corticobasal Degeneration

CurePSP Foundation for PSP/CBD & Related Brain Diseases
Creatine Deficiencies

Association for Creatine Deficiencies 
Cushing’s Disease

Cushing Support & Research Foundation 
Cutaneous Lymphoma

Cutaneous Lymphoma Foundation
Cutis Laxa

Cutis Laxa Internationale 
Cutis Marmorata Telangiectatica Congenita

Cutis Marmorata Telangiectatica Congenita
CMTC-OVM Association, US
Cystic Fibrosis

Cystic Fibrosis Foundation

Cystinosis Foundation
Cystinosis Research Network
Dercum’s Disease

Fat Disorders Research Society, Inc
Desmoid Tumors

Desmoid Tumor Research Foundation 
Dravet Syndrome

Dravet Syndrome Foundation 
Duchenne Muscular Dystrophy

Cure Duchenne
Parent Project Muscular Dystrophy 

Dysautonomia Foundation, Inc.

Dystonia Medical Research Foundation

Dup15q Alliance
Ectodermal Dysplasia

National Foundation for Ectodermal Dysplasias 
Eosinophilia-Myalgia Syndrome

National Eosinophilia-Myalgia Syndrome Network 
Ehlers-Danlos Syndrome

Ehlers-Danlos National Foundation 
Ehlers-Danlos Syndrome Network C.A.R.E.S Inc.

International Association for Food Protein Enterocolitis 
Eosinophilic Disorders

American Partnership for Eosinophilic Disorders 
Epidermolysis Bullosa

Dystrophic Epidermolysis Bullosa Research Association of America (DEBRA)
Erdheim-Chester Disease

ECD Global Alliance

Erythromelalgia Association
Fibrodysplasia Ossificans Progressiva

International Fibrodysplasia Ossificans Progressiva Association
Fibromuscular Dysplasia

Fibromuscular Dysplasia Society of America
FMD Chat
Fibrous Dysplasia

Fibrous Dysplasia Foundation, Inc. 
Fragile X Syndrome

National Fragile X Foundation 
Friedreich’s Ataxia

Friedreich's Ataxia Research Alliance 
Freeman-Sheldon Syndrome

Freeman-Sheldon Research Group, Inc. 
Frontotemporal Degeneration

Association for Frontotemporal Degeneration 
Gastointestinal Motility Disorders (rare)

Association of Gastrointestinal Motility Disorders, Inc.
Gastrointestinal Stromal Tumors

Life Raft Group 
Glycogen Storage Disease

Association for Glycogen Storage Disease 
International Advocate for Glycoprotein Storage Diseases
Guillain-Barré syndrome

GBS/CIDP Foundation International

Hemophilia Federation of America
National Hemophilia Foundation
Hereditary Angioedema

US Hereditary Angioedema Association
Hereditary Hemorrhagic Telangiectasia

Cure HHT Foundation
Hereditary Hemorrhagic Telangiectasia Foundation International, Inc 
Hereditary Leiomyomatosis and Renal Cell Cancer

Hereditary Leiomyomatosis and Renal Cell Cancer Family Alliance 
Hereditary Neuropathy

Hereditary Neuropathy Foundation 
Hermansky-Pudlak Syndrome

Hermansky-Pudlak Syndrome Network Inc.

Histiocytosis Association of America 
Huntington’s Disease

Huntington's Disease Society of America 
Huntington’s Disease Youth Organization

Hydrocephalus Association 
Pediatric Hydrocephalus Foundation

Hypoparathyroidism Association, Inc. 

Soft Bones Foundation 
XLH Network, Inc.
Hypothalamic Hamartoma

Hope for Hypothalamic Hamartomas

Foundation for Ichthyosis & Related Skin Types 
Incontinentia Pigmenti

Incontinentia Pigmenti International Foundation 
Intractable Childhood Epilepsy

Intractable Childhood Epilepsy Alliance 
Iron Disorders (rare)

Iron Disorders Institute
Juvenile Myelomonocytic Leukemia

JMML Foundation
Juvenile Myositis

Cure JM Foundation
Kennedy’s Disease

Kennedy's Disease Association 
Kidney Disease (rare forms)

NephCure Foundation
Klippel-Trenaunay Syndrome

Klippel-Trenaunay Support Group

United Leukodystrophy Foundation 

Fat Disorders Research Society, Inc
Lipomatosis (familial multiple)

Fat Disorders Research Society, Inc
Lowe Syndrome

Lowe Syndrome Association 

LAM Foundation
Lymphangiomatosis & Gorham's Disease Alliance, Inc. 
Lymphatic Malformations

Liam's Land 

National Lymphedema Network 
Lysosomal Acid Lipase Deficiency

LAL Solace, Inc
Macrocephaly-Capillary Malformation

M-CM Network
Madelung’s Disease

Fat Disorders Research Society, Inc
Marfan Syndrome

National Marfan Foundation 
Maple Syrup Urine Disease

MSUD Family Support

Mastocytosis Society 

Melorheostosis Association 

Mesothelioma Applied Research Foundation 
Metabolic Diseases

CLIMB: Children Living with Inherited Metabolic Diseases
Metachromatic Leukodystrophy

MLD Foundation 
Mitochondrial Disease

United Mitochondrial Disease Foundation 
Moebius Syndrome

Moebius Syndrome Foundation

Mucolipidosis Type IV
Mucolipidosis IV Foundation
MPS (Mucopolysaccharidoses)

National MPS Society 
Multiple Endocrine Neoplasia

American Multiple Endocrine Neoplasia Support Hageman Foundation 
Multiple Sclerosis

Consortium of Multiple Sclerosis Centers 
Myalgic Encephalomyelitis

Wisconsin Myalgic Encephalomyelitis/Chronic Fatigue Syndrome Association, Inc 
Myasthenia Gravis

Myasthenia Gravis Foundation of America 

MPN Research Foundation 
Multiple Myeloma

International Myeloma Foundation 

Myocarditis Foundation 

Myositis Association 
Myotonic Dystrophy

Myotonic Dystrophy Foundation
Myotubular Myopathy

Joshua Frase Foundation

Narcolepsy Network, Inc.
Necrotizing Enterocolitis

Morgan Leary Vaughan Fund
Neimann-Pick Disease

National Niemann-Pick Disease Foundation, Inc.
Neurodegeneration with Brain Iron Accumulation

NBIA Disorders Association

Neurofibromatosis, Inc.
Neuromyelitis Optica

Guthy-Jackson Charitable Foundation
NGLY1 Deficient

Grace Wilsey Foundation

Ocular Melanoma

A Cure in Sight
Ocular Melanoma Foundation
Organic Acidemia Metabolic Disorder

Organic Acidemia Association
Osteogenesis Imperfecta

Osteogenesis Imperfecta Foundation

Oxalosis and Hyperoxaluria Foundation
Paget’s Disease

Paget Foundation for Paget's Disease of Bone and Related Disorders
Pancreatic Cancer

Pancreatic Cancer Action Network
Parkinson’s Disease (rare forms)

Parkinson’s Disease Foundation

International Pemphigus Pemphigoid Foundation
Peroxisomal Disorders

Global Foundation for Peroxisomal Disorders
Phelan-McDermid Syndrome

Phelan-McDermid Syndrome Foundation

Children's PKU Network
National PKU Alliance
National PKU News
Polycystic Kidney Disease

PKD Foundation
Platelet Disorders

Platelet Disorder Support Association

American Porphyria Foundation
Prader-Willi Syndrome

Prader-Willi Syndrome Association (USA)
Primary Ciliary Dyskinesia

PCD/Ks Foundation
Primary Immunodeficiency Diseases

Immune Deficiency Foundation
Primary Sclerosing Cholangitis

PSC Partners Seeking a Cure
Progressive Supranuclear Palsy/Corticobasal Degeneration

CurePSP Foundation for PSP/CBD & Related Brain Diseases
Pseudomyxoma Peritonei

PMP Research Foundation
Pulmonary Fibrosis

Pulmonary Fibrosis Foundation
Coalition for Pulmonary Fibrosis
Pulmonary Hypertension

Pulmonary Hypertension Association
Pulmonary Nontuberculous Myobacterial Disease

NTM Information & Research

RASopathies Network
Rare Disease Advocacy Organizations

Ben’s Friends
EMD Serono
Global Genes
Recurrent Respiratory Papillomatosis

Recurrent Respiratory Papillomatosis Foundation
Reflex Sympathetic Dystrophy Syndrome

Reflex Sympathetic Dystrophy Association of America
Rett Syndrome

International Rett Syndrome Foundation
Rett Syndrome Research Trust

Jarod’s Juggernaut
Rothmund-Thomson Syndrome

Rothmund-Thomson Syndrome Foundation
Sanfilippo Syndrome

Jonah’s Just Begun
Red Sanfilippo AC

Sarcoid Networking Association

Sarcoma Foundation of America

Scleroderma Foundation
Scleroderma Research Foundation
Short Bowel Syndrome

Oley Foundation
SBS Cure Project
Short Bowel Syndrome Foundation, Inc.
Shwachman-Diamond Syndrome

Shwachman-Diamond Syndrome Foundation
SLC13AS Deficiency

Tess Foundation
Smith-Magenis Syndrome

Parents and Researchers Interested in Smith-Magenis Syndrome (PRISMS)
Snyder-Robinson Syndrome

Snyder-Robinson Foundation

Sotos Syndrome
Sotos Syndrome Support Association
Spasmodic Dysphonia

National Spasmodic Dysphonia Association
Spasmodic Torticollis

National Spasmodic Torticollis Association
Spastic Paraplegia

Spastic Paraplegia Foundation
Spinal Muscular Atrophy

Children with Spinal Muscular Atrophy Ukraine
Cure SMA
Families of Spinal Muscular Atrophy
Succinic Semialdehyde Dehydrogenase Deficiency

SSADH Association
Sudden Unexplained Death in Childhood

SUDC Foundation
Stevens Johnson Syndrome

Stevens Johnson Syndrome Foundation and Support Group
Sturge-Weber Syndrome

Sturge-Weber Foundation
Syncope (rare forms)

Syncope Trust and Reflex Anoxic Seizures (STARS)

American Syringomyelia & Chiari Alliance Project
Worldwide Syringomyelia & Chiari Task Force
Tarlov Cyst Disease

Tarlov Cyst Disease Foundation
Tay-Sachs Disease

National Tay-Sachs and Allied Diseases Association
Tourette Syndrome (pediatric)

Tourette Syndrome Association, Inc.

MEBO Research, Inc.
Trigeminal Autonomic Cephalgia and Migraine

Cluster Headache Support Group Inc
Trigeminal Neuralgia

TNA - The Facial Pain Association
Tuberous Sclerosis

Tuberous Sclerosis Alliance
Turner Syndrome

Turner Syndrome Society of the United States
Urea Cycle Disorders

National Urea Cycle Disorders Foundation

Vasculitis Foundation
Vestibular Disorders (rare forms)

Vestibular Disorders Association
Von Hippel-Lindau Syndrome

VHL Family Alliance
WAGR Syndrome

International WAGR Syndrome Association
Williams Syndrome

Williams Syndrome Association
Williams Syndrome Family of Hope
Wilson Disease

Wilson Disease Association
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