Rare Disease News and Research

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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

Genmab's HuMax-CD4 receives U.S. orphan drug designation

Genmab's HuMax-CD4 receives U.S. orphan drug designation

Scientists have developed the first mouse model Werner’s syndrome

Scientists have developed the first mouse model Werner’s syndrome

FDA has granted orphan drug designation to Myogen's ambrisentan, for the treatment of pulmonary arterial hypertension

FDA has granted orphan drug designation to Myogen's ambrisentan, for the treatment of pulmonary arterial hypertension

New ethics committee regulations impede and delay clinical research

New ethics committee regulations impede and delay clinical research

No compulsory genetic testing for newborn babies in Europe

No compulsory genetic testing for newborn babies in Europe

Gene mutation gradually causes devastating effects on cellular structure and function in Hutchinson-Gilford progeria syndrome

Gene mutation gradually causes devastating effects on cellular structure and function in Hutchinson-Gilford progeria syndrome

Chemical agents extracted from Caribbean sea squirt may help find cancer treatments for certain deadly tumours

Chemical agents extracted from Caribbean sea squirt may help find cancer treatments for certain deadly tumours