Searching for answers when your child is suffering from a rare or undiagnosed condition can be frustrating. We have organized the information from various websites in an easy-to-find way. While CC4C cannot endorse or guarantee any of these resources, many of our families have found these sites to be useful.
Article: How Patients With Rare Diseases Can Scour The World For A Cure on IDEAS.TED.COM
Center for Genetic and Rare Diseases via National Institutes of Health www.rarediseases.info.NIH.gov
This website for the NIH has links for suspected genetic disorders, tips for the undiagnosed, pages with financial aid, patient advocacy groups, travel and lodging assistance, and all the funded clinical studies in the world that are looking for participants with symptoms to be studied.
Search for trials at www.clinicaltrials.gov or call the Genetic and Rare Disease Call Center line at 1-888-205-2311
National Organization for Rare Disorders www.rarediseases.org or call at 800-999-NORD
NORD has Medication Assistance programs to help pay for medications that may not be covered by insurance, disease specific assistance programs if you already have a diagnosis, travel and lodging assistance for study participants, and a staff of information specialist to answer questions about resources, clinical trials and more.
Rare Genomics Institute http://raregenomics.org/
RGI does genome sequencing and analysis of patient genomes. They do not charge for qualifying patient participants.
SeriousFun Camps https://www.seriousfunnetwork.org/home
At CC4C we know that your child is more than their condition, so we’ve included a link to Serious Fun Camps. These camps were founded by Paul Newman to be a fun place where children with serious illnesses can connect with others like themselves in medically safe environments. There are 30 camps around the country and world.
Travel Assistance Programs
You’ve found the ideal program and doctor for your child, but it’s in another state. How do you get your child to the treatment they need? Below are some organizations that help qualified patients get to treatment.
National Patient Travel Center http://www.patienttravel.org/ or call 1-800-296-1217
Provides information about all forms of charitable, long-distance medical transportation and provides referrals to all appropriate sources of help available in the national charitable medical transportation network to ensure that no ﬁnancially-needy patient is denied access to distant specialized medical evaluation, diagnosis or treatment for lack of a means of long-distance medical transportation.
Mercy Medical Airlift http://mercymedical.org/
Affiliated with National Patient Travel Center but can occasionally help a wider range of patients.
Air Care Alliance http://www.aircareall.org/listings.htm A listing of organizations with volunteers flying to help others. Some of the listed sites are only regional, while some are nation and even worldwide.
Healthcare Hospitality Network, Inc http://www.nahhh.org/
Provides lodging and support services to patients, families and their loved ones who are receiving medical treatment far from home.
Ronald McDonald House Charities http://www.rmhc.org
RMHC helps families stay together during treatment by finding housing that’s near a hospitalized child. They have 290 chapters in 58 countries and regions.
Specific Pages We Found Helpful
NORD’S PATIENT ASSISTANCE PROGRAMS http://www.rarediseases.org/patients-and-families/patient-assistance
This page has several assistance programs which should your child qualify could be useful.
- The Medication Assistance Programs page contains a list of specific medications that might be otherwise unaffordable without help from the program.
- The Disease Specific Assistance Programs are designed to help patients with out-of-pocket cost associated with specific diseases, including premiums, deductibles, and sometimes travel cost, infusions, and diagnostic testing. A list of specific diseases is included on the site.
- The Travel and Lodging Assistance for Study Participants assists patients who are participation in clinical trials with travel and lodging costs incurred during visits to the study sites and provides a list of specific diseases currently in trial.
NORD’S NEWS ABOUT CLINICAL TRIALS http://www.rarediseases.org/patients-and-families/clinical-trials
NORD provides information about opportunities to participate in clinical trials so that patients and their physicians may decide whether specific studies are appropriate for them.
Member List of National Organization for Rare Diseases
Many of these organizations specialize in a specific rare condition. CC4C neither endorses nor recommends any of these organizations, but has made this list available for your convenience.
Acid Maltase Deficiency Association (AMDA)
Acromegaly Community, Inc.
AKU Society of North America
Alagille Syndrome Alliance
Alpha 1 Association
Alpha 1 Foundation
Alport Syndrome Foundation
ALS Association-Greater Philadelphia Chapter
Alternating Hemiplegia of Childhood Foundation
American Autoimmune Related Diseases Association, Inc.
American Behcet’s Disease Association
American Brain Tumor Association
American Partnership for Eosinophilic Disorders
American Porphyria Foundation
American Self-Help Group Clearinghouse
American Syringomyelia Alliance Project, Inc.
Amyloidosis Support Groups, Inc
Aplastic Anemia & MDS International Foundation, Inc.
Association for Creatine Deficiencies
Association for Frontotemporal Degeneration (AFTD)
Association for Glycogen Storage Disease
Association of Gastrointestinal Motility Disorders, Inc. (AGMD)
Association of Genetic Support of Australasia Inc
A-T Children’s Project
Basal Cell Carcinoma Nevus Syndrome Life Support Network
Batten Disease Support and Research Association
Benign Essential Blepharospasm Research Foundation, Inc.
Breath of Hope Inc
CADASIL Association, Inc.
Canadian Organization for Rare Disorders (CORD)
Caring for Carcinoid Foundation
Cerebral Palsy Symptoms
Children with Spinal Muscular Atrophy Ukraine
Children’s Cardiomyopathy Foundation
Children’s Craniofacial Association
Children’s PKU Network
Children’s Tumor Foundation
Chromosome 18 Registry and Research Society
Chromosome Disorder Outreach, Inc.
Chronic Granulomatous Disease Association, Inc.
Cicatricial Alopecia Research Foundation
Cleft Palate Foundation
CLOVES Syndrome Community
CMTC-OVM Cutis Marmorata Telangiectatica Congenita
Coalition for Pulmonary Fibrosis
Congenital Hyperinsulinism International
Consortium of Multiple Sclerosis Centers
Contact A Family
Cornelia de Lange Syndrome Foundation Inc.
Council for Bile Acid Deficiency Diseases
CurePSP Foundation for PSP/CBD & Related Brain Diseases
Cushing Support and Research Foundation
Cutaneous Lymphoma Foundation
Cutis Laxa Internationale
Cystinosis Research Network, Inc.
Desmoid Tumor Reseach Foundation
Dysautonomia Foundation, Inc.
Dystonia Medical Research Foundation
DEBRA of America (Dystrophic Epidermolysis Bullosa Research Association of America)
ECD Global Alliance
Ehlers-Danlos National Foundation
(The) Erythromelalgia Association
Families of Spinal Muscular Atrophy
Family Caregiver Alliance
Family Support Network of North Carolina
Fibromuscular Dysplasia Society of America
Fibrous Dysplasia Foundation
Foundation Fighting Blindness
Foundation for Ichthyosis & Related Skin Types
Friedreich’s Ataxia Research Alliance (FARA)
Freeman-Sheldon Research Group, Inc.
GBS/CIDP Foundation International
Genetic & Rare Disorders Organisation
Global Foundation for Peroxisomal Disorders
Guthy Jackson Charitable Foundation
Hemophilia Federation of America
Hereditary Disease Foundation, Inc.
Hereditary Hemorrhagic Telangiectasia (HHT) Foundation International
Hereditary Leiomyomatosis and Renal Cell Cancer Family Alliance
Hereditary Neuropathy Foundation
Histiocytosis Association, Inc.
Hope for Hypothalamic Harmartomas
Huntington’s Disease Society of America
Hypoparathyroidism Association, Inc
Immune Deficiency Foundation
Incontinentia Pigmenti International Foundation
In Need of Diagnosis, Inc (INOD)
International Association for Food Protein Entercolitis
International Fibrodysplasia Ossificans Progressiva (FOP) Association
International Foundation for CDKL5 Research
International Myeloma Foundation
International Pemphigus Pemphigoid Foundation (IPPF)
International Rett Syndrome Foundation
International WAGR Syndrome Association
Intractable Childhood Epilepsy Alliance (ICE Epilepsy Alliance)
Iron Disorder Institute
Jack McGovern Coats Disease Foundation
(The) JMML Foundation
Kennedy’s Disease Association
(The) Klippel-Trenaunay Support Group
LAL Solace, Inc.
(The) LAM Foundation
Les Turner ALS Foundation, Ltd.
Liam’s Land Organization, Inc.
Life Raft Group
Locks of Love
Lowe Syndrome Association
Lymphangiomatosis & Gorham’s Disease Alliance, Inc
(The) Mastocytosis Society, Inc.
MEBO Research, Inc.
Mercy Medical Airlift
Mesothelioma Applied Research Foundation
Mesothelioma Prognosis Network
MPN Research Foundation
Mucolipidosis Type IV Foundation, Inc (ML4)
Moebius Syndrome Foundation
Myasthenia Gravis Foundation of America
(The) Myocarditis Foundation
Myotonic Dystrophy Foundation
Narcolepsy Network, Inc.
National Adrenal Diseases Foundation
National Alopecia Areata Foundation
National Ataxia Foundation
National Brain Tumor Society
National Eosinophilia Myalgia Syndrome Network
National Foundation for Ectodermal Dysplasias
National Fragile X Foundation
National Hemophilia Foundation
National Lymphedema Network
National Marfan Foundation
National MPS (Mucopolysaccharidoses/Mucolipdoses) Society
National Multiple Sclerosis Society
National Niemann-Pick Disease Foundation, Inc.
National Organization for Albinism and Hypopigmentation, NOAH
National PKU Alliance
National PKU News
National Spasmodic Dysphonia Association
National Spasmodic Torticollis Association
National Tay-Sachs and Allied Diseases Association, Inc.
National Urea Cycle Disorders Foundation
NBIA Disorders Association
(The) NephCure Foundation
Organic Acidemia Association
Osteogenesis Imperfecta Foundation
(The) Oxalosis and Hyperoxaluria Foundation
Paget Foundation for Paget’s Disease of Bone and Related Disorders
Pancreatic Cancer Action Network
Parent Project Muscular Dystrophy
Parent to Parent of New Zealand, Inc.
Parkinson’s Disease Foundation, Inc.
Phelan-McDermid Syndrome Foundation
Platelet Disorder Support Association
PMP Research Foundation
Prader-Willi Syndrome Association (USA)
PRISMS (Parents & Researchers Interested in Smith-Magenis Syndrome)
Proyecto Pide un Deseo Mexico
PSC Partners Seeking A Cure (Primary Sclerosing Cholangitis)
Pulmonary Fibrosis Foundation
Pulmonary Hypertension Association
Recurrent Respiratory Papillomatosis Foundation
Red Sanfilippo A.C.
Reflex Sympathetic Dystrophy Syndrome Association of America
Sarcoid Networking Association
Sarcoma Foundation of America
Scleroderma Foundation, Inc.
Scleroderma Research Foundation
(The) Short Bowel Syndrome Foundation, Inc
Shwachman-Diamond Syndrome Foundation
Soft Bones, Inc.
Sotos Syndrome Support Association
Spastic Paraplegia Foundation
Stevens Johnson Syndrome Foundation and Support Group
Syncope Trust & Reflex Anoxic Seizures-STARS
Taiwan Foundation For Rare Disorders
TNA – The Facial Pain Association
Tourette Syndrome Association, Inc.
Tuberous Sclerosis Alliance
Turner Syndrome Society of the United States
United Leukodystrophy Foundation
United Mitochondrial Disease Foundation
US Hereditary Angioedema Association
Vestibular Disorders Association, VEDA
VHL Family Alliance
(The) Williams Syndrome Association
Wilson’s Disease Association
Wisconsin Myalgic Encephalomyelitis/Chronic Fatigue Syndrome Association, Inc.