Rare Disease News and Research

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FDA grants Vical orphan-drug designation

FDA grants Vical orphan-drug designation

Infants with Krabbe disease saved by cord blood stem cells

Infants with Krabbe disease saved by cord blood stem cells

Zevalin safe and effective for non-Hogdkin's lymphoma patients

Zevalin safe and effective for non-Hogdkin's lymphoma patients

Myogen's Ambrisentan granted orphan drug designation by Europeans

Myogen's Ambrisentan granted orphan drug designation by Europeans

9/11 rescue workers screening program to continue

9/11 rescue workers screening program to continue

Fifteen-year hunt uncovers gene behind pseudothalidomide syndrome

Fifteen-year hunt uncovers gene behind pseudothalidomide syndrome

The liver may produce most of the body's good cholesterol

The liver may produce most of the body's good cholesterol

First UK cases of previously rare disease reported in gay men

First UK cases of previously rare disease reported in gay men

Onconase granted orphan drug status for malignant mesothelioma in Australia

Onconase granted orphan drug status for malignant mesothelioma in Australia

Three different leukemias are all caused by mutations that alter a specific enzyme controlling blood cell proliferation

Three different leukemias are all caused by mutations that alter a specific enzyme controlling blood cell proliferation

Marburg Virus responsible for disease outbreak in Angola

Marburg Virus responsible for disease outbreak in Angola

New center focuses on rare Batten disease

New center focuses on rare Batten disease

Common clinical problem, Rosacea

Common clinical problem, Rosacea

FDA approval of Vaccinia Immune Globulin to treat adverse reactions to smallpox vaccination

FDA approval of Vaccinia Immune Globulin to treat adverse reactions to smallpox vaccination

Pharmaceutical and biotechnology industry in the new EU countries presents considerable potential

Pharmaceutical and biotechnology industry in the new EU countries presents considerable potential

Acute eosinophilic pneumonia occurring at higher than normal rate among U.S. troops in Iraq

Acute eosinophilic pneumonia occurring at higher than normal rate among U.S. troops in Iraq

Vitamin E supplements may reduce the risk for death from amyotrophic lateral sclerosis (ALS)

Vitamin E supplements may reduce the risk for death from amyotrophic lateral sclerosis (ALS)

Cancer vaccine based on listeria bacteria shows promise

Cancer vaccine based on listeria bacteria shows promise

Researchers reveal potential new role for Glivec/Imatinib

Researchers reveal potential new role for Glivec/Imatinib

Titanium rib becomes 1st new FDA-approved spine deformity treatment in 40 years

Titanium rib becomes 1st new FDA-approved spine deformity treatment in 40 years

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