Support Organizations
Education
Caregiver Support
Cell & Gene Medicine Information
Listed By Disease
1p36 Deletion Syndrome
1p36 Deletion Support & Awareness
Achalasia
Martin Mueller IV Achalasia Awareness Foundation
Acid Maltase Deficiency (see Pompe’s Disease)
ADCY5-induced Dyskinesia
Adipose Tissue Disorders
Fat Disorders Resource Society (FDRS)
Adrenal Insufficiency
Adrenoleukodystrophy (ALD)
ADNP Syndrome (Helsmoortel – Van Der Aa Syndrome)
The ADNP Kids Research Foundation
Adult Polyglucosan Body Disease (APBD)
The APBD Research Foundation (APBDRF)
Adrenal Diseases
National Adrenal Diseases Foundation (NADF)
Alagille Syndrome (ALGS)
The Alagille Syndrome Alliance (ALGSA)
Alopecia Areata
National Alopecia Areata Foundation (NAAF)
Alport Syndrome (see Kidney)
Alzheimers
Amyloidosis
Amyloidosis Research Consortium, Inc.
Amyloidosis Support Groups (ASG)
Amyotrophic Lateral Sclerosis
Angelman Syndrome (AS)
Foundation for Angelman Syndrome Therapeutics (FAST)
Aniridia (see WAGR Syndrome)
Antitrypsin Deficiency
Aplastic Anemia
The Aplastic Anemia & MDS International Foundation, Inc., (AA & MDS)
Julia’s Wings Foundation (JWF)
Arterial Tortuosity Syndrome
Arthritis
Asthma
Asthma & Allergy Foundation of America
Ataxia
National Ataxia Foundation (NAF)
Ataxia-telangiectasia
Autism
Autoimmune Disease
Autoinflammatory Diseases
Bannayan-Riley-Ruvalcaba Syndrome (BRRS)
PTEN Hamartoma Tumor Syndrome Foundation
Barth Syndrome
Batten Disease
Batten Disease Support and Research Association (BDSRA)
Charlotte + Gwenyth Gray Foundation
Behçet’s Disease
The American Behçet’s Disease Association (ABDA)
Blindness
Curing Retinal Blindness Foundation
Jack McGovern Coats’ Disease Foundation
Bone Marrow Failure Disorders
American Society of Hematology
Aplastic Anemia and MDS International Foundation
Diamond Blackfan Anemia Foundation
Dyskeratosis Congenita Outreach
Bohring-Opitz Syndrome
Bohring-Opitz Syndrome Foundation, Inc.
Brain
International FOXG1 Foundation
CADASIL
Cancer
Accelerate Brain Cancer Cure (ABC2)
Fibrolamellar Cancer Foundation (FCF)
National Breast Cancer Foundation, Inc.
National Lung Cancer Partnership
National Ovarian Cancer Coalition
Neuroendocrine Tumor Research Foundation (NTRF)
Cardio-Facio-Cutaneous Syndrome
Cardio-Facio-Cutaneous International
Castleman Disease
Castleman Disease Collaborative Network (CDCN)
CDKL5 Disorders
International Foundation for CDKL5 Research (IFCR)
Centronuclear Myopathy
Cerebral Palsy
CFC Syndrome
Charcot-Marie-Tooth (CMT)
Charcot-Marie-Tooth Association (CMTA)
Hereditary Neuropathy Foundation
Chromosome 15q Duplication Syndrome
Chromosome 18
Chromosome 18 Registry & Research Society
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
GBS/CIDP Foundation International
Complex Regional Pain Syndrome (CRPS)
Reflex Sympathetic Dystrophy Syndrome Association (RSDSA)
Costello Syndrome
Cowden Syndrome (CS)
PTEN Hamartoma Tumor Syndrome Foundation
Cushing’s Disease
Cushing’s Support and Research Foundation, Inc. (CSRF)
Cystic Fibrosis
Cystinosis
Cystinosis Research Network (CRN)
Diabetes
Juvenile Diabetes Research Foundation (JDRF)
Dercum’s Disease
Fat Disorders Resource Society (FDRS)
Desmoid Tumor
Desmoid Tumor Research Foundation (DTRF)
DHPS
Down Syndrome
National Down Syndrome Society
Dravet Syndrome (see Epilespsy)
Dyskeratosis Congenita
Eating Disorders
National Eating Disorders Association
Ectodermal Dysplasias
National Foundation for Ectodermal Dysplasias (NFED)
Ehlers-Danlos Syndromes
Eosinophilic Disorders
The American Partnership for Eosinophilic Disorders (APFED)
Epilepsy
Epidermylosis Bullosa (EB) Debra International
Dravet Syndrome Foundation (DSF)
Erdheim-Chester disease (ECD)
Erythromelalgia (EM)
Face | Cranium (Head) Disfigurement or Injury
National Craniofacial Association
Children’s Craniofacial Association
Familial Dysautonomia
Familial Dysautonomia Foundation
Fancomi Anemia
Fibrodysplasia Ossificans Progressiva (FOP)
International Fibrodysplasia Ossificans Progressiva Association (IFOPA)
Fibromuscular Dysplasia
Fibromuscular Dysplasia Society of America (FMDSA)
Fibrous Dysplasia (FD)
Fibrous Dysplasia Foundation, Inc. (FDF)
Food Allergies
Food Allergy Research & Education (FARE)
International Association for Food Protein Enterocolitis (IAFFPE)
FOXG1
International FOXG1 Foundation
Friedreich’s Ataxia
Friedreich’s Ataxia Research Alliance (FARA)
Galactosemia
Gastrointestinal Disorders
International Association for Food Protein Enterocolitis (IAFFPE)
Glycogen Storage Disease
The Association for Glycogen Storage Disease (AGSD)
Gorham’s Disease
Lymphangiomatosis & Gorham’s Disease (LGD) Alliance
Guillain-Barré Syndrome
GBS/CIDP Foundation International
Heart
Children’s Cardiomyopathy Foundation (CCF)
National Coalition for Women with Heart Disease
Hemophagocytic Lymphohistiocytosis (HLH)
Hemophilia
Hemophilia Federation of America
National Hemophilia Foundation
World Federation of Hemophilia
Hemorrhagic Telangiectasia (HHT) – Osler Weber Rendu Syndrome
Cure HHT Foundation International, Inc.
Hereditary Leiomyomatosis and Renal Cell Cancer (HLRCC)
I’m Too Young for This! Cancer Foundation
International Neuroendocrine Cancer Alliance
LiFraumeni Syndrome (LFS) Association
Hermansky-Pudlak Syndrome
Hermansky-Pudlak Syndrome (HPS) Network
Homocystinurias
HSAN1E
Hydrocephalus
Hyper IgM
Hypoglycemia
Hyperinsulinemic hypoglycemia of infancy (PHHI)
Congenital Hyperinsulinism International (CHI)
Hypophosphatasia (HPP)
Soft Bones, Inc.: The US Hypophosphatasia Foundation
Immune Thrombocytopenia (ITP)
Platelet Disorder Support Association (PDSA)
Immunodeficiency and Autoimmune Diseases
Advocacy & Awareness for Immune Disorders Association (AAIDA)
Incontinentia Pigmenti (IP)
National Incontinentia Pigmenti Foundation
Inflammatory Myopathies
The Myositis Association (TMA)
Kabuki Syndrome
KAT6A Syndrome
Kennedy’s Disease
Kernicterus
Parents of Infants and Children with Kernicterus (PICK)
Kidney
Alport Syndrome
The Alport Syndrome Foundation (ASF)
Nephcure Kidney International Foundation
KIF1A Disorder
Klippel-Trenaunay Syndrome
Klippel-Trenaunay Support Group
Krabbe Disease
Lactose Intolerance
Lowe Syndrome
Lowe Syndrome Association, Inc. (LSA)
Leukodystrophy
Leukoencephalopathy with brainstem and spinal cord involvement and lactate elevation (LBSL)
LiFraumeni Syndrome
LiFraumeni Syndrome (LFS) Association
Lipidema
Fat Disorders Resource Society (FDRS)
Liver
Lung
Lupus
Lymphangiomatosis
Lymphangiomatosis & Gorham’s Disease (LGD) Alliance
Lysosomal Storage Disease
Cystinosis Research Network (CRN)
ML4 Mucolipidosis Type IV Foundation
The International Advocate for Glycoprotein Storage Diseases
Macrocephaly-Capillary Malformation (M-CM)
Maple Syrup Urine Disease
Marfan Syndrome
McCune-Albright Syndrome (FD/MAS)
Fibrous Dysplasia Foundation, Inc. (FDF)
Melorheostosis
Mental Illness
Depression and Bipolar Support Alliance
National Alliance on Mental Illness (NAMI)
Mitochondrial Disease
Mitochondrial Disease Action Committee | Mito Action
United Mitochondrial Disease Foundation
Moebius Syndrome
Mowat-Wilson Syndrome
Mucopolysaccharidoses (MPS) | Mucolipidoses (ML)
Multiple Endocrine Neoplasia
The American Multiple Endocrine Neoplasia Support
Multiple Sclerosis
Consortium of Multiple Sclerosis Centers
National Multiple Sclerosis Society
Multiple System Atrophy
Muscular Dystrophy
Duchenne and Becker Muscular Dystrophy
Muscular Dystrophy Association
Parent Project Muscular Dystrophy
Myasthenia Gravis
Myasthenia Gravis Foundation of America, Inc. (MGFA)
Myelodysplastic Syndromes (MDS)
The Aplastic Anemia & MDS International Foundation, Inc., (AA & MDS)
Myeloproliferative Neoplasms (MPNs)
Myotonic Dystrophy
Myotonic Dystrophy Foundation (MDF)
Myotubular Myopathy
N-Glycanase Deficiency
Necrotizing Enterocolitis (NEC)
Neurodegenerative Disorders
Charlotte + Gwenyth Gray Foundation
Neurofibromatosis
Children’s Tumor Foundation (CTF)
Neuropathy
Hereditary Neuropathy Foundation
Nervous System
GBS/CIDP Foundation International
Neurofibromatosis
Niemann-Pick Disease (NPD)
National Niemann-Pick Disease Foundation, Inc.
Noonan Syndromes
Ocular Melanoma
The Aplastic Anemia & MDS International Foundation, Inc., (AA & MDS)
The Rare Cancer Research Foundation
Okur-Chung Neurodevelopmental Syndrome
Opsoclonus Myoclonus Syndrome
Organic Acidemia
Osler Weber Rendu Syndrome (see Hemorrhagic Telangiectasia)
Osteogenesis imperfecta (OI)
Osteogenesis Imperfecta Foundation (OIF)
Osteoporosis
National Osteoporosis Foundation
Parkinson’s Disease
Paraganglioma
Phenylketonuria (PKU)
Prader-Willi Syndrome
Prader-Willi Syndrome Association (USA) (PWSA)
Primary Ciliary Dyskinesia
Perthes
PHACE Syndrome
Phelan-McDermid Syndrome
Phelan-McDermid Syndrome Foundation
Phenylketonuria (PKU)
Pheochromocytoma
Pompe’s Disease
The Acid Maltase Deficiency Association, Inc. (AMDA)
Porphyria
The American Porphyria Foundation (APF)
Proteus Syndrome
PTEN Hamartoma Tumor Syndrome Foundation
Psoriasis
PTEN Syndromes
PTEN Hamartoma Tumor Syndrome Foundation
Pulmonary Fibrosis
Pulmonary Fibrosis Foundation (PFF)
Parkinson’s Disease
European Parkinson’s Disease Association (EPDA)
Parkinson’s Disease Foundation
Peroxisome Biogenesis Disorders – Zellweger Spectrum Disorder (PBD-ZSD)
Global Foundation for Peroxisomal Disorders (GFPD)
Pfeiffer Syndrome
Prader-Willi Syndrome
Foundation for Prader-Willi Research
Primary Biliary Cholangitis
Primary Lateral Sclerosis (PLS)
Spastic Paraplegia Foundation (SPF)
Primary Sclerosing Cholangitis (PSC)
PURA Syndrome
Rare Diseases
Canadian Organization for Rare Disorders (CORD)
Chloe’s Fight Rare Disease Foundation
Indian Organization for Rare Diseases
National Organization for Rare Diseases (NORD)
Organization for Rare Diseases India
Rare and Undiagnosed Network (RUN)
Taiwan Foundation for Rare Disorders
RASopathies
Recurrent Respiratory Papillomatosis
Recurrent Respiratory Papillomatosis Foundation (RRPF)
Reflex Sympathetic Dystrophy Syndrome
Reflex Sympathetic Dystrophy Syndrome Association (RSDSA)
Rett Syndrome
International Rett Syndrome Foundation
Rett Syndrome Research Trust (RSRT)
Rothmund-Thomson Syndrome
Rothmund-Thomson Syndrome Foundation
RYR-1
Sacral Agenesis | Caudal Regression Syndrome
International Sacral Agenesis/Caudal Regression Association
Sanfilippo Syndrome
Sanfilippo Children’s Foundation
Sarcoidosis
Foundation for Sarcoidosis Research
Scleroderma
Scleroderma Research Foundation
SETBP1 Disorder
Short Bowel Syndrome
Shwachman-Diamond Syndrome
Shwachman-Diamond Syndrome Foundation (SDSF)
Sickle Cell Disease
American Sickle Cell Anemia Association (ASCAA)
Association de Lutte Contre la Drépanocytose
Foundation for Sickle Cell Disease Research
Global Sickle Cell Disease Network
Sickle Cell Disease Association of America
Sitosterolema
Skin
Ichthyosis
Foundation for Ichthyosis & Related Skin Types (FIRST)
International Pemphigus & Pemphigoid Foundation
National Incontinentia Pigmenti Foundation
SLC13A5 | Citrate Transporter Disorder
Smith-Magenis Syndrome (SMS)
PRISMS (Parents & Researchers Interested in Smith-Magenis Syndrome)
Snyder-Robinson Syndrome (SRS)
Spasmodic Dysphonia
National Spasmodic Dysphonia Association (NSDA)
Spastic Parapalegia
Spastic Paraplegia Foundation (SPF)
Spina Bifida
Spina Bifida Association (SBA)
The Gwendolyn Strong Foundation (theGSF)
Succinic Semialdehyde Dehydrogenase Deficiency (SSADH)
SYNGAP mutations
Bridge the Gap – SYNGAP Education and Research Foundation
TANGO2 Mutations
Tay-Sachs
National Tay-Sachs & Allied Diseases Association, Inc. (NTSAD)
Telomere Biology Disorders
Thalassemia
TK2 Deficiency
Trimethylaminuria
Urea Cycle Disorders
National Urea Cycle Disorders Foundation (NUCDF)
USP7 Deletion | Mutation
Foundation for USP-7 Related Diseases
Van Lohuizen Syndrome
WAGR Syndrome
International WAGR Syndrome Association
Waldenstrom’s Macroglobulinemia (WM)
International Waldenstrom’s Macroglobulinemia Foundation (IWMF)
Wilm’s Tumor (see WAGR Syndrome)
Zellweger Syndrome