Disease Information from NORD, National Organization for Rare Disorders, Inc.

• Database Subscriptions

Many libraries, schools, universities, and hospitals subscribe to NORD’s Rare Disease Database for unlimited access to reports on more than 1,150 diseases.

• Index of Rare Diseases

This is the list of diseases currently covered in the Rare Disease Database.

• Rare Disease Database

Search this database for reports on more than 1,150 diseases.

View sample report

• Index of Organizations

This is the list of organizations in NORD’s Organizational Database.

• Organizational Database

Read about more than 2,000 patient organizations and other sources of help.

NORD's
Washington Office
Read about events on Capitol Hill, funding for rare-disease research, and other topics of interest from NORD's office in Washington, DC.

Programs & Services

NORD’s Patient Assistance Programs

Since 1987, NORD has administered programs to assist uninsured or under-insured individuals in securing life-saving or life-sustaining medications. In addition to the estimated 50 million Americans who have no health insurance, an increasing number of insured individuals have policies that do not reimburse for prescription drugs. Others have policies with low annual caps on prescription drug expenditures.

NORD works closely with humanitarian-minded pharmaceutical and biotechnology companies to ensure that certain vital medications are available to those individuals whose income is too high to qualify for Medicaid but too low to pay for their prescribed medications. NORD’s programs have set the standards for fairness, equity, and unbiased eligibility. The NORD programs have gained respect from the patient communities, pharmaceutical companies, healthcare professionals, government officials, and the public. Participants have assurance that NORD protects their confidentiality. There is no conflict of interest with respect to NORD’s eligibility decisions.

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Now Available: NORD Medical Foods Assistance Program for Patients with Phenylketonuria (PKU)

The National Organization for Rare Disorders (NORD) Medical Foods Assistance Program for Patients with Phenylketonuria (PKU) is now accepting applications for financial support. The program is a new safety net that provides financial support for PKU patients without benefit coverage to obtain their preferred medical food (limited to amino acid modified products that contain a synthetic source of protein and exclude phenylalanine). Funding is limited and patients must meet the following criteria:

Diagnosis of PKU via written attestation by physician
Residents of the United States and its territories
Documentation demonstrating exhaustion of available state assistance and/or denial of insurance coverage for medical foods
Income up to 500% of Federal Poverty Level (based on family size)

To apply for the program, please download this form, fill it out, and fax it to 203-798-2964, or mail it to:

NORD
55 Kenosia Avenue
Danbury, CT 06813

To obtain more information on the NORD Medical Foods (PKU) Program, call 1-877-798-8716.

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Currently, NORD administers programs for:

Acorda Therapeutics
Zanaflex Capsules™ Uninsured Individual Program
(Tizanidine Hydrochloride)
Conditions:
Spasticity
Contact:
Reimbursement Assistance Program at 1-800-464-0097
ATON PHARMA
ATON PHARMA Patient Assistance Program
Cuprimine, Demser, Edecrin, Lacrisert, Mephyton, Syprine
Conditions:
Wilson’s Disease, Rheumatoid Arthritis, Cystinuria, pheochromocytoma, edema associated with congestive heart failure, cirrhosis of the liver, renal disease, idiopathic edema, lymphedema; dry eye syndromes, vitamin k deficiency
Contact:
The ATON Call Center toll-free at 1-877-286-6549
BioMarin Pharmaceuticals
Naglazyme Premium/Co-Payment Assistance Program For Privately Insured Individuals
(galsulfase)
Conditions:
Mucopolysaccharidosis Type VI (Maroteaux-Lamy Syndrome)
Contact:
1-800-999-6673
Cephalon, Inc.
TRISENOX® Patient Assistance Program
(arsenic trioxide) injection
Conditions:
relapsed or refractory acute promyelocytic Leukemia (APL), Multiple Myeloma
Contact:
Cephalon Oncology Reimbursement Expertise, 1-866-261-7730
Cephalon, Inc.
PROVIGIL® Assistance Program
(modafinil)
Conditions:
Narcolepsy
Contact:
the Provigil® Reimbursement Assistance Hotline at 1-800-675-8415
Medtronic, Inc.
ITB® Therapy Patient Assistance Program
Lioresal® Intrathecal (baclofen injection)
Conditions:
Severe spasticity
Contact:
NORD at 1-800-999-6673 e-mail ealmonte@rarediseases.org
Jazz Pharmaceutical, Inc.
XYREM® Patient Assistance Program
(sodium oxybate)
Conditions:
Narcolepsy with cataplexy
Contact:
Xyrem Success Program at 1-866-997-3688
Questcor Pharmaceuticals, Inc.
ACTHAR® GEL Patient Assistance Program
(ACTH)
Conditions:
Infantile spasms, multiple sclerosis, Crohns disease, Adrenal insufficiency, etc.
Contact:
Acthar Support & Access Program at 1-888-435-2284
Rare Disease Therapeutics, Inc.
CYSTADANE® Patient Assistance Program
(betaine anhydrous for oral solution)
Conditions:
Homocystinuria
Contact:
NORD at 1-800-999-6673 e-mail nvondle@rarediseases.org
Rare Disease Therapeutics, Inc.
ORFADIN® Patient Assistance Program
(nitisinone)
Conditions:
Hereditary Tyrosinemia Type I
Contact:
NovaFactor at 1-888-454-8860
Sanofi - Aventis
RILUTEK® Patient Assistance Program
(riluzole)
Conditions:
Amyotrophic lateral sclerosis
Contact:
NORD at 1-800-459-7599 or e-mail ealmonte@rarediseases.org
Sigma-tau Pharmaceuticals, Inc.
CARNITOR® Drug Assistance Program
(L-Carnitine or levo carnitine)
Conditions:
Primary systemic carnitine deficiency, dialysis, chronic fatigue syndrome (CFS), several rare metabolic disorders
Contact:
NORD at 1-800-999-6673 e-mail nvondle@rarediseases.org
Sigma-tau Pharmaceuticals, Inc.
MATULANE® Drug Assistance Program
(procarbazine hydrochloride)
Conditions:
Hodgkins disease (Stage III and IV) and certain other lymphomas
Contact:
NORD at 1-800-999-6673 e-mail nvondle@rarediseases.org
Teva Neuroscience, Inc.
COPAXONE® Medication Assistance Program
(glatiramer acetate, for injection)
Conditions:
Multiple sclerosis (relapsing-remitting)
Contact:
Shared Solutions at 1-800-887-8100
Teva Pharmaceuticals Inc. USA
Tev-Tropin® Patient Assistance Program
Tev-Tropin® Co-Payment Assistance Program For Privately Insured Individuals
Somatropin (rDNAorigin) for injection
Conditions:
growth failure due to inadequate secretion of normal endogenous growth hormone
Contact:
Growth Solutions at 1-866-838-8767
Ucyclyd Pharma, Inc.
BUPHENYL®
(sodium phenylbuterate)
Conditions:
Urea cycle disorders
Contact:
Ucyclyd Reimbursement Hotline at 1-800-711-0811
NORD MS Premium /Co-Payment Assistance Program
Conditions:
Multiple Sclerosis (MS)
Contact:
1-800-634-7207
NORD CAPS Premium & Co-payment Assistance Fund
Conditions:
Cryopyrin-associated periodic syndromes
Contact:
1-800-999-6673 x 1224
NORD Cervical Dystonia Co-Payment Assistance Program
Conditions:
Cervical Dystonia (Spasmodic Torticollis)
Contact:
1-800-999-6673 x 1311
NORD CPP Premium /Co-Payment Assistance Program
Conditions:
Chronic Plaque Psoriasis (CPP)
Contact:
1-800-634-7207
NORD Intrathecal Therapy for Pain Management Co-Payment Assistance Program
Conditions:
Intrathecal Therapy for Pain Management
Contact:
1-888-774-2581
NORD Advanced Renal Cell Carcinoma Co-Payment Assistance Program
Conditions:
Advanced Renal Cell Carcinoma
Contact:
1-866-828-8902
NORD Hodgkins Lymphoma Co-Payment Assistance Program
Conditions:
Hodgkins Lymphoma
Contact:
1-800-999-6673
NORD Chronic Non-Infectious Uveitis Co-Payment Assistance Fund
Conditions:
Non-infectious uveitis
Contact:
1-800-999-6673
NORD Hunter Syndrome (MPS II) Premium/Co-Payment Assistance Program
Conditions:
Hunter Syndrome (MPS II)
Contact:
1-800-999-6673
NORD MPS VI Premium/Co-Payment Assistance Program
Conditions:
mucopolysaccharidosis Type VI (Maroteaux-Lamy Syndrome)
Contact:
1-800-999-6673
NORD Nephrotic Syndrome Premium/Co-Payment Assistance Program
Condition:
Nephrotic Syndrome
Contact:
1-888-435-2284
NORD PNH Premium/Co-Payment Assistance Program
Conditions:
Paroxysmal Nocturnal Hemoglobinuria
Contact: 1-800-999-6673
NORD Infantile Spasms Premium/Co-Payment Assistance Program
Conditions:
Infantile spasms/West Syndrome
Contact:
NORD at 1-888-435-2284
NORD Opsoclonus Myoclonus Premium/Co-Payment Assistance Program
Conditions:
Opsoclonus Myoclonus
Contact: NORD at 1-888-435-2284
NORD Hepatocellular Carcinoma Co-Payment Assistance Program
Conditions:
Hepatocellular Carcinoma
Contact: 1-866-828-8902
NORD Phenylketonuria (PKU) Premium/Co-Payment Assistance Program
Conditions:
Phenylketonuria
Contact: 1-866-924-0100
MS Related Acute Exacerbation Premium/Co-Payment Assistance Program
Conditions:
MS related acute exacerbation
Contact: 1-888-435-2284

NORD also administers Early Access Programs for investigational new drugs (INDs) under the Food and Drug Administration’s approved “Treatment-IND” programs that allow for a limited number of individuals to receive certain investigational drugs prior to FDA marketing approval, as well as a Travel Assistance Program that provides travel and lodging assistance for patients participating in clinical and research studies.

For information about the programs listed on this page, please use the contact information associated with that particular program or write to: PatientAssistance@rarediseases.org.

ABOUT | CONTACT NORD

Since 1983, working toward the prevention, treatment, and cure of rare “orphan” diseases.

NORD Resource Guide

The new and expanded NORD Resource Guide (5th edition) is now available. It lists more than 1,300 organizations helping people with rare disorders.

Travel Assistance

For information about free or low-cost travel to treatment facilities or sites of clinical studies, NORD refers patients and families to the National Patient Travel Center.

Become a NORD Member

As a member of NORD, you’ll receive benefits in addition to the satisfaction of knowing you’re helping people with rare diseases. Read about our levels of Membership and the benefits of each.

Last modified Friday, January 30, 2009