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.
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Research
Primary Liver Cancer
This study is designed to determine the safety and effectiveness of MTC-DOX
in treating individuals with primary liver cancer. MTC-DOX is Doxorubicin or
DOX, a chemotherapy drug, bound to tiny, microscopic particles of iron and carbon
(MTCs). Magnetic properties of these particles make it possible to direct MTC-DOX
to specific tumor sites in the liver by placing a magnet on the body surface.
It is hoped that MTC-DOX, used with the magnet, may target the chemotherapy
directly to liver tumors and provide a treatment for patients with primary liver
cancer.
Currently, there is no approved chemotherapy drug specifically for primary
liver cancer, although Doxorubicin is an approved chemotherapy drug. A control
group of patients will receive Doxorubicin administered intravenously.
The treatments will be administered every three weeks through six treatment
cycles. The study will be conducted at several sites in North America, Asia,
and Europe, and will involve approximately 240 participants.
For information, contact Dave Donahue, Associate Director of Clinical Development,
FeRx Inc., 9171 Towne Centre Drive, Suite 575 San Diego, CA 92122 (telephone:
858 677-7788; e-mail: ddonahue@ferx.com).
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Over 1200 Disease Reports
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Since 1983, working toward the prevention, treatment, and cure of rare
“orphan” diseases.
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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
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