Rare Disease News and Research

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Paving the way for future pan-European clinical trials

Paving the way for future pan-European clinical trials

Completion of first clinical trial of Apatone for cancer treatment

Completion of first clinical trial of Apatone for cancer treatment

Discovery of new pathway causing cell death related to frontotemporal dementia and ALS

Discovery of new pathway causing cell death related to frontotemporal dementia and ALS

Rochester receives $1.2 million to battle rare childhood disease

Rochester receives $1.2 million to battle rare childhood disease

Mutations in the insulin gene can cause permanent neonatal diabetes

Mutations in the insulin gene can cause permanent neonatal diabetes

Muscadine grape skin extract shows promise for prostate cancer

Muscadine grape skin extract shows promise for prostate cancer

Fruit flies have a taste for carbonated water

Fruit flies have a taste for carbonated water

Symposium on children born with clubfoot

Symposium on children born with clubfoot

FDA approves Somatuline Depot to treat acromegaly

FDA approves Somatuline Depot to treat acromegaly

FDA grants orphan drug designation to TREANDA

FDA grants orphan drug designation to TREANDA

New Cockayne syndrome discovery

New Cockayne syndrome discovery

Pharmion and MethylGene announce orphan drug designation for treatment of Hodgkin's lymphoma

Pharmion and MethylGene announce orphan drug designation for treatment of Hodgkin's lymphoma

Vaccines approved by the FDA are at least a million times safer than the viruses that they prevent

Vaccines approved by the FDA are at least a million times safer than the viruses that they prevent

Gluten-free foods - growing market - taste issues

Gluten-free foods - growing market - taste issues

New understanding of Sly Syndrome

New understanding of Sly Syndrome

Higher child leukaemia rates near nuclear facilities

Higher child leukaemia rates near nuclear facilities

Higher leukaemia rates in children living near nuclear facilities

Higher leukaemia rates in children living near nuclear facilities

Discovery of enzyme critical in DNA replication

Discovery of enzyme critical in DNA replication

Goltz syndrome gene found

Goltz syndrome gene found

breakthrough in the treatment of idiopathic pulmonary haemosiderosis

breakthrough in the treatment of idiopathic pulmonary haemosiderosis