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New findings identify Sortilin as a potential therapeutic site to alter PGRN pathology in dementia

New findings identify Sortilin as a potential therapeutic site to alter PGRN pathology in dementia

New simple blood test may help diagnose Niemann-Pick type C disease

New simple blood test may help diagnose Niemann-Pick type C disease

Alpha-synuclein protein in Parkinson's disease inhibits autophagy: Research

Alpha-synuclein protein in Parkinson's disease inhibits autophagy: Research

BioMarin's BMN-701 receives FDA orphan drug designation for treatment of Pompe disease

BioMarin's BMN-701 receives FDA orphan drug designation for treatment of Pompe disease

BioMarin Pharmaceutical acquires ZyStor Therapeutics, furthers development of ZC-701 for Pompe disease

BioMarin Pharmaceutical acquires ZyStor Therapeutics, furthers development of ZC-701 for Pompe disease

Scientists develop novel chemotherapeutic method for anticancer drug delivery

Scientists develop novel chemotherapeutic method for anticancer drug delivery

Researchers discover potential method for neurodegenerative disease detection

Researchers discover potential method for neurodegenerative disease detection

Enzyme mTOR regulates autophagy: Study

Enzyme mTOR regulates autophagy: Study

New research focuses on calcium channels in lysosome membrane

New research focuses on calcium channels in lysosome membrane

Elevation of Smurf1 may play a role in molecular basis of PAH: Study

Elevation of Smurf1 may play a role in molecular basis of PAH: Study

Disorder in degradation of proteins and other macromolecules in cells leads to serious diseases

Disorder in degradation of proteins and other macromolecules in cells leads to serious diseases

Accumulation of mutated protein may explain damaging cellular behavior in Huntington's disease

Accumulation of mutated protein may explain damaging cellular behavior in Huntington's disease

Raptor Pharmaceutical to present DR Cysteamine trial data at Lysosomal Disease Network Symposium

Raptor Pharmaceutical to present DR Cysteamine trial data at Lysosomal Disease Network Symposium

Research offers new hope for rare metabolic disorder

Research offers new hope for rare metabolic disorder

Results from Phase 2a clinical trial of Raptor Pharmaceutical's cysteamine bitartrate published

Results from Phase 2a clinical trial of Raptor Pharmaceutical's cysteamine bitartrate published

IKK phosphorylate: A double-edged sword in Huntington's disease

IKK phosphorylate: A double-edged sword in Huntington's disease

Nanoprobes deliver drugs directly to the right tumor cellular compartments

Nanoprobes deliver drugs directly to the right tumor cellular compartments

Albert Einstein College of Medicine receives SU2C grant for cancer research

Albert Einstein College of Medicine receives SU2C grant for cancer research

Lipid that accumulates in brain cells drives cell death, says research

Lipid that accumulates in brain cells drives cell death, says research

Study suggests a new improved treatment option for children with Hurler's syndrome

Study suggests a new improved treatment option for children with Hurler's syndrome