Resources

Resources

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

Nationwide Organizations

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)

Cardio-Facio-Cutaneous International

Caring for Carcinoid Foundation

Cerebral Palsy Symptoms

Charcot-Marie-Tooth Association

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

CLIMB

CLOVES Syndrome Community

CMTC-OVM Cutis Marmorata Telangiectatica Congenita

CMTC-OVM-US

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 Foundation

Cystinosis Research Network, Inc.

Desmoid Tumor Reseach Foundation

Dravet.org

Dysautonomia Foundation, Inc.

Dystonia Medical Research Foundation

DEBRA of America (Dystrophic Epidermolysis Bullosa Research Association of America)

Dup15 Alliance

ECD Global Alliance

Ehlers-Danlos National Foundation

(The) Erythromelalgia Association

EURORDIS

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 Alliance

Genetic & Rare Disorders Organisation

Global Foundation for Peroxisomal Disorders

Guthy Jackson Charitable Foundation

Hageman 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

Hydrocephalus Association

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.

M-CM Network

MEBO Research, Inc.

Melorheostosis Association

Mercy Medical Airlift

Mesothelioma Applied Research Foundation

Mesothelioma Prognosis Network

Mesothelioma Resource

MitoAction

MPN Research Foundation

Mucolipidosis Type IV Foundation, Inc (ML4)

Moebius Syndrome Foundation

Myasthenia Gravis Foundation of America

(The) Myocarditis Foundation

Myositis Association

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

Neurofibromatosis, Inc.

NOMID Alliance

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.

PCD/KS Foundation

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

SSADH Association

Stevens Johnson Syndrome Foundation and Support Group

Sturge-Weber Foundation

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

Vasculitis Foundation

Vestibular Disorders Association, VEDA

VHL Family Alliance

(The) Williams Syndrome Association

Wilson’s Disease Association

Wisconsin Myalgic Encephalomyelitis/Chronic Fatigue Syndrome Association, Inc.