Gaucher's Disease News and Research

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Gaucher disease is an inherited metabolic disorder in which harmful quantities of a fatty substance called glucocerebroside accumulate in the spleen, liver, lungs, bone marrow, and sometimes in the brain.
Study finds disease-causing mutation in French-Canadians

Study finds disease-causing mutation in French-Canadians

Opportunities and risks of direct-to-consumer genetic health risk tests

Opportunities and risks of direct-to-consumer genetic health risk tests

FDA authorizes marketing of first direct-to-consumer GHR tests for certain diseases

FDA authorizes marketing of first direct-to-consumer GHR tests for certain diseases

Scientists propose potential new treatment for Gaucher disease

Scientists propose potential new treatment for Gaucher disease

URMC researchers identify potential new means of treating severe genetic diseases in children

URMC researchers identify potential new means of treating severe genetic diseases in children

New study finds genetic link between Parkinson's disease and cognitive decline

New study finds genetic link between Parkinson's disease and cognitive decline

Pathology of Krabbe's disease may provide insights on late-onset neurodegenerative diseases

Pathology of Krabbe's disease may provide insights on late-onset neurodegenerative diseases

Enzyme deficiency in Krabbe's disease may point to new mechanisms underlying Parkinson's disease

Enzyme deficiency in Krabbe's disease may point to new mechanisms underlying Parkinson's disease

Yale Cancer Center researchers identify cause of myeloma

Yale Cancer Center researchers identify cause of myeloma

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

Mutation that increases sphingolipid levels can lead to neurodegeneration

Mutation that increases sphingolipid levels can lead to neurodegeneration

Robert G. Miller awarded $1.5 million grant to help fund Neuraltus' Phase 2 clinical study of NP001 for ALS

Robert G. Miller awarded $1.5 million grant to help fund Neuraltus' Phase 2 clinical study of NP001 for ALS

CORD calls upon governments, others to join forces to make Canada's Rare Disease Strategy a reality

CORD calls upon governments, others to join forces to make Canada's Rare Disease Strategy a reality

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Eliglustat drug improves liver, spleen size and hemoglobin level in adults with Gaucher disease type 1

Eliglustat drug improves liver, spleen size and hemoglobin level in adults with Gaucher disease type 1

AT2101 drug, evaluated for Gaucher disease, slows progression of Parkinson's disease in mice

AT2101 drug, evaluated for Gaucher disease, slows progression of Parkinson's disease in mice

Gaucher disease: an interview with Dr Clement Olivier, Shire

Gaucher disease: an interview with Dr Clement Olivier, Shire

European scientists set up new therapeutic approaches to tackle gene defects

European scientists set up new therapeutic approaches to tackle gene defects

Research findings may offer new therapeutic target for management of Gaucher diseas

Research findings may offer new therapeutic target for management of Gaucher diseas

Genzyme granted Priority Review for Cerdelga investigational oral therapy for Gaucher disease

Genzyme granted Priority Review for Cerdelga investigational oral therapy for Gaucher disease