Rare Disease Report
Physicians
Physicians
Patients & Caregivers

Appendix B: Patient Advocacy Groups

A
 
Achalasia

Martin Mueller IV Achalasia Awareness Foundation
 
Acid Maltese Deficiency

Acid Maltase Deficiency Association (AMDA)
 
Acromegaly

Acromegaly Community, Inc.
 
Adult Polyglucosan Body Disease

Adult Polyglucosan Body Disease Research Foundation
APBD Research Foundation

AKU
AKU Society of North America
 
Adrenal Diseases

National Adrenal Diseases Foundation
 
Alagille Syndrome

Alagille Syndrome Alliance
 
Albinism

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

Amyloidosis Support Groups, Inc
 
Aplastic Anemia

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

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

American Autoimmune Related Diseases Association, Inc.
 
Autoinflammatory Diseases

NOMID Alliance
 
B
 
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
 
C
 
Cadasil

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
 
CDKL5

International Foundation for CDKL5 Research
 
Charlot-Marie-Tooth Disease

Charcot-Marie-Tooth Association 
 
Cholangiocarcinoma

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

Cystinosis Foundation
Cystinosis Research Network
 
D
 
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

Dysautonomia Foundation, Inc.
 
Dystonia

Dystonia Medical Research Foundation
 
Dup15q

Dup15q Alliance
 
E
 
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.
 
Enterocolitis

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
 
Erthromelalgia

Erythromelalgia Association
 
F
 
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 
 
G
 
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
 
H
 
Hemophilia

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

Histiocytosis Association of America 
 
Huntington’s Disease

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

Hydrocephalus Association 
Pediatric Hydrocephalus Foundation
 
Hypoparathyroidism

Hypoparathyroidism Association, Inc. 
 
Hypophosphatasia

Soft Bones Foundation 
XLH Network, Inc.
 
Hypothalamic Hamartoma

Hope for Hypothalamic Hamartomas
 
I
 
Ichthyosis

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
 
J
 
Juvenile Myelomonocytic Leukemia

JMML Foundation
 
Juvenile Myositis

Cure JM Foundation
 
K
 
Kennedy’s Disease

Kennedy's Disease Association 
 
Kidney Disease (rare forms)

NephCure Foundation
 
Klippel-Trenaunay Syndrome

Klippel-Trenaunay Support Group
 
L
 
Leukodystrophy

United Leukodystrophy Foundation 
 
Lipedema

Fat Disorders Research Society, Inc
 
Lipomatosis (familial multiple)

Fat Disorders Research Society, Inc
 
Lowe Syndrome

Lowe Syndrome Association 
 
Lymphangiomatosis 

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

Liam's Land 
 
Lymphedema

National Lymphedema Network 
 
Lysosomal Acid Lipase Deficiency

LAL Solace, Inc
 
M
 
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

Mastocytosis Society 
 
Melorheostosis

Melorheostosis Association 
 
Mesothelioma

Mesothelioma Applied Research Foundation 
 
Metabolic Diseases

CLIMB: Children Living with Inherited Metabolic Diseases
 
Metachromatic Leukodystrophy

MLD Foundation 
 
Mitochondrial Disease

MitoAction
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 
 
Myelofibrosis

MPN Research Foundation 
 
Multiple Myeloma

International Myeloma Foundation 
 
Myocarditis

Myocarditis Foundation 
 
Myositis

Myositis Association 
 
Myotonic Dystrophy

Myotonic Dystrophy Foundation
 
Myotubular Myopathy

Joshua Frase Foundation
 
 
N
 
Narcolepsy

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

Neurofibromatosis, Inc.
 
Neuromyelitis Optica

Guthy-Jackson Charitable Foundation
 
NGLY1 Deficient

Grace Wilsey Foundation
 
O

Ocular Melanoma

A Cure in Sight
Ocular Melanoma Foundation
 
Organic Acidemia Metabolic Disorder

Organic Acidemia Association
 
Osteogenesis Imperfecta

Osteogenesis Imperfecta Foundation
 
Oxalosis

Oxalosis and Hyperoxaluria Foundation
 
P
 
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
 
Pemphigus/Pemphigoid

International Pemphigus Pemphigoid Foundation
 
Peroxisomal Disorders

Global Foundation for Peroxisomal Disorders
 
Phelan-McDermid Syndrome

Phelan-McDermid Syndrome Foundation
 
Phenylketonuria

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

PKD Foundation
 
Platelet Disorders

Platelet Disorder Support Association
 
Porphyria

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
 
R
 
RASopathies

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
 
Rhambomyosarcoma

Jarod’s Juggernaut
 
Rothmund-Thomson Syndrome

Rothmund-Thomson Syndrome Foundation
 
 
S
 
Sanfilippo Syndrome

Jonah’s Just Begun
Red Sanfilippo AC
 
Sarcoidosis

Sarcoid Networking Association
 
Sarcoma

Sarcoma Foundation of America
 
Scleroderma

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)
 
Syringomyelia

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

Tarlov Cyst Disease Foundation
 
Tay-Sachs Disease

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

Tourette Syndrome Association, Inc.
 
Trimethylaminuria

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
 
U
 
Urea Cycle Disorders

National Urea Cycle Disorders Foundation
 
V
 
Vasculitis

Vasculitis Foundation
 
Vestibular Disorders (rare forms)

Vestibular Disorders Association
 
Von Hippel-Lindau Syndrome

VHL Family Alliance
 
W
 
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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