Lysosomal Storage Disorders News and Research

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New research suggests another possible mechanism for Alzheimer’s

New research suggests another possible mechanism for Alzheimer’s

Rare inherited enzyme disorder offers new clues about fibrotic conditions

Rare inherited enzyme disorder offers new clues about fibrotic conditions

UVA researchers make unexpected discovery about irreplaceable nerve cells

UVA researchers make unexpected discovery about irreplaceable nerve cells

New therapeutic approach may delay neurodegeneration in rare genetic disease

New therapeutic approach may delay neurodegeneration in rare genetic disease

Researchers describe important step toward gene therapy for patients with Sandhoff disease

Researchers describe important step toward gene therapy for patients with Sandhoff disease

Study points at lysosomal storage disorder genes as potential key contributors to Parkinson's

Study points at lysosomal storage disorder genes as potential key contributors to Parkinson's

Shire and Parion Sciences to collaborate on P-321 for ophthalmic indications

Shire and Parion Sciences to collaborate on P-321 for ophthalmic indications

Scientists discover treatment that improves symptoms in mouse model of juvenile Batten disease

Scientists discover treatment that improves symptoms in mouse model of juvenile Batten disease

FDA grants permission to market new Seeker System for screening rare metabolic disorders in newborns

FDA grants permission to market new Seeker System for screening rare metabolic disorders in newborns

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

Novel drug delivery system may offer new hope for patients with rare fatal genetic disorder

Novel drug delivery system may offer new hope for patients with rare fatal genetic disorder

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

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

CHOP researchers delay symptoms, extend lifespan in animal model of Batten disease

CHOP researchers delay symptoms, extend lifespan in animal model of Batten disease

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

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

State highlights: Calif.'s Prop. 45 fight; new benefits for same sex couples in N.C.; Aetna sheds jobs after Medicaid drop

State highlights: Calif.'s Prop. 45 fight; new benefits for same sex couples in N.C.; Aetna sheds jobs after Medicaid drop

European scientists set up new therapeutic approaches to tackle gene defects

European scientists set up new therapeutic approaches to tackle gene defects

Expert in gene therapy joins The Children's Hospital of Philadelphia

Expert in gene therapy joins The Children's Hospital of Philadelphia

Levels of certain fats in cerebral spinal fluid can predict patients with HIV, say scientists

Levels of certain fats in cerebral spinal fluid can predict patients with HIV, say scientists

Genzyme marks 2013 International Rare Disease Day

Genzyme marks 2013 International Rare Disease Day

Researchers discover key interaction at heart of promising new treatment for Fabry disease

Researchers discover key interaction at heart of promising new treatment for Fabry disease