Niemann Pick Disease News and Research

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Small clinical trial shows promise for treating rare neurodegenerative disease

Small clinical trial shows promise for treating rare neurodegenerative disease

Gene therapy may be effective method for treating Niemann-Pick disease, type C1

Gene therapy may be effective method for treating Niemann-Pick disease, type C1

Newborn screen for Niemann-Pick disease type C ready for piloting

Newborn screen for Niemann-Pick disease type C ready for piloting

Simple test for measuring bile acids in biological fluids can help diagnose severe fat storage disorder

Simple test for measuring bile acids in biological fluids can help diagnose severe fat storage disorder

New method could help scientists better predict disease-causing mutations in people's genes

New method could help scientists better predict disease-causing mutations in people's genes

Mutation that increases sphingolipid levels can lead to neurodegeneration

Mutation that increases sphingolipid levels can lead to neurodegeneration

Vtesse reports preliminary results from VTS-270 Phase 1 trial for treatment of Niemann-Pick Disease Type C

Vtesse reports preliminary results from VTS-270 Phase 1 trial for treatment of Niemann-Pick Disease Type C

Scientists identify molecular 'lock' that enables Ebola virus to gain entry to cells

Scientists identify molecular 'lock' that enables Ebola virus to gain entry to cells

TSRI study examines body’s own response against chronic protein misfolding

TSRI study examines body’s own response against chronic protein misfolding

Cholesterol plays vital role in cell mobility and tissue invasion

Cholesterol plays vital role in cell mobility and tissue invasion

New dual-pronged approach for treating Niemann-Pick type C disease

New dual-pronged approach for treating Niemann-Pick type C disease

Clinical trial to evaluate cyclodextrin against Niemann-Pick disease type C1

Clinical trial to evaluate cyclodextrin against Niemann-Pick disease type C1

Study reveals technique that allows scientists to watch lipid metabolism in live zebrafish

Study reveals technique that allows scientists to watch lipid metabolism in live zebrafish

Potential new way to treat rare inherited metabolic disorders

Potential new way to treat rare inherited metabolic disorders

PerkinElmer Genetics launches new Lysosomal Storage Disorder panel

PerkinElmer Genetics launches new Lysosomal Storage Disorder panel

Scientists identify cellular protein that plays a critical role in Ebola virus infection

Scientists identify cellular protein that plays a critical role in Ebola virus infection

Scientists identify critical protein responsible for Ebola virus infection

Scientists identify critical protein responsible for Ebola virus infection

Researchers discover NPC1 protein as crucial for Ebola virus to enter host cells

Researchers discover NPC1 protein as crucial for Ebola virus to enter host cells

PCA-Rx represents next generation of chelation technology for ASD patients

PCA-Rx represents next generation of chelation technology for ASD patients

Genetic Disorder Tracker introduced by Guidepoint Global

Genetic Disorder Tracker introduced by Guidepoint Global