The National Organization for Rare Disorders (NORD)

Database Subscriptions

Many libraries, schools, universities, and hospitals subscribe to NORD’s Rare Disease Database for unlimited access to reports on more than 1,200 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 1,200 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

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

 

Research

Hereditary Angioedema

Baxter Healthcare manufactured a C-1 inhibitor protein, extracted from human blood and heat-sterilized, for HAE in Europe. However, this treatment, which was administered intravenously, is no longer available. Aventis now distributes a plasma-based product in Europe and is making it available on a limited basis in the United States. For information about obtaining the Aventis product, patients and physicians should contact the Immune Deficiency Foundation in Maryland:

Immune Deficiency Foundation
40 West Chesapeake Avenue
Suite 308
Towson, MD 21230
Telephone: (410) 321-6647
Toll Free: (800) 296-4433
e-mail: jb@primaryimmune.org
Web site: http://www.primaryimmune.org

Dyax Corporation is developing a recombinant form of the HAE C-1 inhibitor called DX-88 for the treatment of patients who have acute, episodic attacks of HAE. Clinical trials of this biotechnology product are underway. This product does not contain human plasma. Dyax refers to the study as EDEMA 1 (Evaluation of DX-88’s Effect on Mitigating Angioedema). Treatment centers are being established (December 2002) in the United States and worldwide. Patients 10 years of age or older who suffer from acute attacks of HAE are eligible for inclusion in the trial. Participants will be administered one dose by intravenous infusion, and will be followed for a period of 28 days.

In the United States, patients and parents or guardians of patients may call an automated call center at 866-814-4915 to register for the EDEMA 1 trial. For additional information, contact:

Clinical Operations
Dyax Corp.
300 Technology Square
Cambridge, MA 02139
Telephone: (617) 225-2500

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ABOUT | CONTACT NORD

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

Previous Clinical Trials
These studies were listed earlier on this page. Contact the researchers if you would like to know whether they are still recruiting.

CADASIL
Pelizaeus-Merzbacher Disease
Platelet Disorders
ARVC/ARVD
Immune Thrombocytopenic Purpura
Sturge-Weber Syndrome
Brain Metastases Originating from Breast Cancer
Duplication 12p
Rheumatoid Arthritis/Juvenile Rheumatoic Arthritis, Lupus, Scleroderma or Myositis
Moyamoya Disease
Prader-Willi Syndrome
Larsen Syndrome Registry and Web Site
Primary Liver Cancer
Risk Factors in Autoimmune Disease
Neuroendocrine Unit Studies at MGH
Congenital Lactic Acidosis
Hereditary Angioedema
NINDS Physicians Study Fabry's Disease
Inherited Bone Marrow Failure Disorders
Genzyme Recruiting Patients for Dose Frequency Study
Fabry Disease
Primary Ciliary Dyskinesia
Neurofibromatosis Type I
Primary Lateral Sclerosis
FENIB
Alkaptonuria
Behcet's Disease
Infantile Neuronal Ceroid Lipofuscinosis (INCL)
Turner Syndrome


Free Booklets For Physicians

Free Booklets For PhysiciansNORD offers free booklets for physicians and other medical profes-
sionals. To request copies, or learn how topics are selected,
click here
.

Networking Program

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Database Subscriptions

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Last modified Thursday, December 10, 2009