Targeting the GBA1 pathway to slow Parkinson disease: Insights into clinical aspects, pathogenic mechanisms and new therapeutic avenues

Classification of GBA1 Variants in Parkinson's Disease: The GBA1 ‐PD Browser

(2023) Sitki Cem Parlar et al.   MOVEMENT DISORDERS

Ambroxol reverses tau and α-synuclein accumulation in a cholinergic N370S GBA1 mutation model

(2022) Shi Yu Yang et al.   HUMAN MOLECULAR GENETICS

Lysosomal ceramides regulate Cathepsin B-mediated processing of saposin C and glucocerebrosidase activity

(2022) Myung Jong Kim et al.   HUMAN MOLECULAR GENETICS

No Evidence That Glucosylsphingosine Is a Biomarker for Parkinson’s Disease: Statistical Differences Do Not Necessarily Indicate Biological Significance

(2022) Adenrele Gleason et al.   MOVEMENT DISORDERS

Recombinant pro-CTSD (cathepsin D) enhances SNCA/α-Synuclein degradation in α-Synucleinopathy models

(2022) Susy Prieto Huarcaya et al.   Autophagy

Misfolded GBA/β-glucocerebrosidase impairs ER-quality control by chaperone-mediated autophagy in Parkinson disease

(2022) Sheng-Han Kuo et al.   Autophagy

Increased Stroke Risk in Patients with Parkinson's Disease with LRRK2 Mutations

(2022) Sara Grisanti et al.   MOVEMENT DISORDERS

Glucocerebrosidase-associated Parkinson disease: Pathogenic mechanisms and potential drug treatments

(2022) Matthew E. Gegg et al.   NEUROBIOLOGY OF DISEASE

Genetic heterogeneity on sleep disorders in Parkinson’s disease: a systematic review and meta-analysis

(2022) Jingxuan Huang et al.   Translational Neurodegeneration

Genetic variations in GBA1 and LRRK2 genes: Biochemical and clinical consequences in Parkinson disease

(2022) Laura J. Smith et al.   Frontiers in Neurology

Prosaposin Reduces α-Synuclein in Cells and Saposin C Dislodges it from Glucosylceramide-enriched Lipid Membranes

(2022) Rika Kojima et al.   JOURNAL OF MOLECULAR NEUROSCIENCE

Lysosomal lipid alterations caused by glucocerebrosidase deficiency promote lysosomal dysfunction, chaperone-mediated-autophagy deficiency, and alpha-synuclein pathology

(2022) Alba Navarro-Romero et al.   npj Parkinsons Disease

Neuronopathic GBA1L444P Mutation Accelerates Glucosylsphingosine Levels and Formation of Hippocampal Alpha-Synuclein Inclusions

(2022) Casey L. Mahoney-Crane et al.   JOURNAL OF NEUROSCIENCE

A randomized single and multiple ascending dose study in healthy volunteers of LTI‐291, a centrally penetrant glucocerebrosidase activator

(2021) Jonas M. Heijer et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Profiling the Biochemical Signature of GBA‐Related Parkinson's Disease in Peripheral Blood Mononuclear Cells

(2021) Micol Avenali et al.   MOVEMENT DISORDERS

The Mutation Matters: CSF Profiles of GCase , Sphingolipids, α‐Synuclein in PD GBA

(2021) Stefanie Lerche et al.   MOVEMENT DISORDERS

Altered ceramide metabolism is a feature in the extracellular vesicle-mediated spread of alpha-synuclein in Lewy body disorders

(2021) Marzena Kurzawa-Akanbi et al.   ACTA NEUROPATHOLOGICA

Lewy Body–like Inclusions in Human Midbrain Organoids Carrying Glucocerebrosidase and α‐Synuclein Mutations

(2021) Junghyun Jo et al.   ANNALS OF NEUROLOGY

Glucocerebrosidase Activity is not Associated with Parkinson's Disease Risk or Severity

(2021) Nurit Omer et al.   MOVEMENT DISORDERS

Serum lipid profile among sporadic and familial forms of Parkinson’s disease

(2021) Daniel Macías-García et al.   npj Parkinsons Disease

Plasma Glucosylsphingosine in GBA1 Mutation Carriers with and without Parkinson's Disease

(2021) Matthew Surface et al.   MOVEMENT DISORDERS

Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model

(2021) Catherine Viel et al.   Scientific Reports

Safety, Pharmacokinetics, and Pharmacodynamics of Oral Venglustat in Patients with Parkinson’s Disease and a GBA Mutation: Results from Part 1 of the Randomized, Double-Blinded, Placebo-Controlled MOVES-PD Trial

(2021) M. Judith Peterschmitt et al.   Journal of Parkinsons Disease

Direct targeting of wild-type glucocerebrosidase by antipsychotic quetiapine improves pathogenic phenotypes in Parkinson’s disease models

(2021) Lena F. Burbulla et al.   JCI Insight

Sphingolipid changes in Parkinson L444P GBA mutation fibroblasts promote α-synuclein aggregation

(2021) Céline Galvagnion et al.   BRAIN

Gut-seeded α-synuclein fibrils promote gut dysfunction and brain pathology specifically in aged mice

(2020) Collin Challis et al.   NATURE NEUROSCIENCE

Ambroxol for the Treatment of Patients With Parkinson Disease With and Without Glucocerebrosidase Gene Mutations

(2020) Stephen Mullin et al.   JAMA Neurology

Glucocerebrosidase deficiency promotes release of α-synuclein fibrils from cultured neurons

(2020) Matthew E Gegg et al.   HUMAN MOLECULAR GENETICS

Impact of GBA1 variants on long-term clinical progression and mortality in incident Parkinson’s disease

(2020) Thomas B Stoker et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Substrate reduction therapy using Genz‐667161 reduces levels of pathogenic components in a mouse model of neuronopathic forms of Gaucher disease

(2020) Shani Blumenreich et al.   JOURNAL OF NEUROCHEMISTRY

Evaluating the Role of SNCA , LRRK2 , and GBA in Chinese Patients With Early‐Onset Parkinson's Disease

(2020) Yongping Chen et al.   MOVEMENT DISORDERS

High Glucosylceramides and Low Anandamide Contribute to Sensory Loss and Pain in Parkinson's Disease

(2020) Katharina Klatt‐Schreiner et al.   MOVEMENT DISORDERS

GBA ‐Related Parkinson's Disease: Dissection of Genotype–Phenotype Correlates in a Large Italian Cohort

(2020) Simona Petrucci et al.   MOVEMENT DISORDERS

GBA variants in REM sleep behavior disorder: a multicenter study

(2020) Lynne Krohn et al.   NEUROLOGY

β-Glucocerebrosidase activity in GBA-linked Parkinson disease

(2020) Young Eun Huh et al.   NEUROLOGY

Pro-cathepsin D, Prosaposin, and Progranulin: Lysosomal Networks in Parkinsonism

(2020) Nahid Tayebi et al.   TRENDS IN MOLECULAR MEDICINE

Ambroxol as a novel disease-modifying treatment for Parkinson’s disease dementia: protocol for a single-centre, randomized, double-blind, placebo-controlled trial

(2019) C. R. A. Silveira et al.   BMC Neurology

Insights into GBA Parkinson's disease pathology and therapy with induced pluripotent stem cell model systems

(2019) Pascale Baden et al.   NEUROBIOLOGY OF DISEASE

Glucocerebrosidase and its relevance to Parkinson disease

(2019) Jenny Do et al.   Molecular Neurodegeneration

Brain-First versus Gut-First Parkinson’s Disease: A Hypothesis

(2019) Per Borghammer et al.   Journal of Parkinsons Disease

Ambroxol Hydrochloride Improves Motor Functions and Extends Survival in a Mouse Model of Familial Amyotrophic Lateral Sclerosis

(2019) Alexandra Bouscary et al.   Frontiers in Pharmacology

Pharmacologic properties of high-dose ambroxol in four patients with Gaucher disease and myoclonic epilepsy

(2019) Yoon-Myung Kim et al.   JOURNAL OF MEDICAL GENETICS

Reduced sphingolipid hydrolase activities, substrate accumulation and ganglioside decline in Parkinson’s disease

(2019) Mylene Huebecker et al.   Molecular Neurodegeneration

Parkinson's Disease: Glucocerebrosidase 1 Mutation Severity Is Associated with CSF Alpha‐Synuclein Profiles

(2019) Stefanie Lerche et al.   MOVEMENT DISORDERS

Glucocerebrosidase activity, cathepsin D and monomeric α-synuclein interactions in a stem cell derived neuronal model of a PD associated GBA1 mutation

(2019) Shi-yu Yang et al.   NEUROBIOLOGY OF DISEASE

Cholesterol and multilamellar bodies: Lysosomal dysfunction in GBA-Parkinson disease

(2018) Patricia García-Sanz et al.   Autophagy

Acid ceramidase inhibition ameliorates α-synuclein accumulation upon loss of GBA1 function

(2018) Myung Jong Kim et al.   HUMAN MOLECULAR GENETICS

Features ofGBA-associated Parkinson’s disease at presentation in the UKTracking Parkinson’sstudy

(2018) Naveed Malek et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Characterization of Brain Lysosomal Activities in GBA-Related and Sporadic Parkinson’s Disease and Dementia with Lewy Bodies

(2018) Tim E. Moors et al.   MOLECULAR NEUROBIOLOGY

Fetal gene therapy for neurodegenerative disease of infants

(2018) Giulia Massaro et al.   NATURE MEDICINE

Parkinson's disease phenotype is influenced by the severity of the mutations in the GBA gene

(2018) Avner Thaler et al.   PARKINSONISM & RELATED DISORDERS

The Parkinson Pandemic—A Call to Action

(2018) E. Ray Dorsey et al.   JAMA Neurology

Integrated Genetic Analysis of Racial Differences of Common GBA Variants in Parkinson's Disease: A Meta-Analysis

(2018) Yuan Zhang et al.   Frontiers in Molecular Neuroscience

Chaperone-mediated autophagy as a therapeutic target for Parkinson disease

(2018) Philip Campbell et al.   EXPERT OPINION ON THERAPEUTIC TARGETS

Higher serum cholesterol and decreased Parkinson's disease risk: A statin-free cohort study

(2018) Violetta Rozani et al.   MOVEMENT DISORDERS

Effects of ambroxol on the autophagy-lysosome pathway and mitochondria in primary cortical neurons

(2018) J. Magalhaes et al.   Scientific Reports

Lifetime risk of common neurological diseases in the elderly population

(2018) Silvan Licher et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Global, regional, and national burden of Parkinson's disease, 1990–2016: a systematic analysis for the Global Burden of Disease Study 2016

(2018) E. Ray Dorsey et al.   LANCET NEUROLOGY

Survival rates among Parkinson's disease patients who carry mutations in the LRRK2 and GBA genes

(2018) Avner Thaler et al.   MOVEMENT DISORDERS

Glucocerebrosidase mutations and synucleinopathies: Toward a model of precision medicine

(2018) Fabio Blandini et al.   MOVEMENT DISORDERS

The Emerging Evidence of the Parkinson Pandemic

(2018) E. Ray Dorsey et al.   Journal of Parkinsons Disease

The heat shock protein amplifier arimoclomol improves refolding, maturation and lysosomal activity of glucocerebrosidase

(2018) Cathrine K. Fog et al.   EBioMedicine

Insights into the structural biology of Gaucher disease

(2017) Laura Smith et al.   EXPERIMENTAL NEUROLOGY

Alpha-synuclein induces the unfolded protein response in Parkinson’s disease SNCA triplication iPSC-derived neurons

(2017) Sabrina M Heman-Ackah et al.   HUMAN MOLECULAR GENETICS

GBA Analysis in Next-Generation Era

(2017) Stefania Zampieri et al.   JOURNAL OF MOLECULAR DIAGNOSTICS

Glucosylsphingosine Promotes α-Synuclein Pathology in Mutant GBA-Associated Parkinson's Disease

(2017) Yumiko V. Taguchi et al.   JOURNAL OF NEUROSCIENCE

AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy

(2017) Giuseppe Morabito et al.   MOLECULAR THERAPY

Cerebrospinal fluid β-glucocerebrosidase activity is reduced in parkinson's disease patients

(2017) Lucilla Parnetti et al.   MOVEMENT DISORDERS

N370S-GBA1 mutation causes lysosomal cholesterol accumulation in Parkinson's disease

(2017) Patricia García-Sanz et al.   MOVEMENT DISORDERS

Oligomeric α-synuclein and glucocerebrosidase activity levels in GBA-associated Parkinson’s disease

(2017) S. Pchelina et al.   NEUROSCIENCE LETTERS

Serum lipid alterations in GBA -associated Parkinson's disease

(2017) Leonor Correia Guedes et al.   PARKINSONISM & RELATED DISORDERS

Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models

(2017) S. Pablo Sardi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Oral ambroxol increases brain glucocerebrosidase activity in a nonhuman primate

(2017) Anna Migdalska-Richards et al.   SYNAPSE

The Role of Lipids Interacting with α-Synuclein in the Pathogenesis of Parkinson’s Disease

(2017) Céline Galvagnion   Journal of Parkinsons Disease

A Human Neural Crest Stem Cell-Derived Dopaminergic Neuronal Model Recapitulates Biochemical Abnormalities in GBA1 Mutation Carriers

(2017) Shi-Yu Yang et al.   Stem Cell Reports

Ambroxol effects in glucocerebrosidase and α-synuclein transgenic mice

(2016) Anna Migdalska-Richards et al.   ANNALS OF NEUROLOGY

Specifically neuropathic Gaucher's mutations accelerate cognitive decline in Parkinson's

(2016) Ganqiang Liu et al.   ANNALS OF NEUROLOGY

Survival and dementia inGBA-associated Parkinson's disease: The mutation matters

(2016) Roberto Cilia et al.   ANNALS OF NEUROLOGY

Endoplasmic reticulum and lysosomal Ca2+ stores are remodelled in GBA1-linked Parkinson disease patient fibroblasts

(2016) Bethan S. Kilpatrick et al.   CELL CALCIUM

Progress and potential of non-inhibitory small molecule chaperones for the treatment of Gaucher disease and its implications for Parkinson disease

(2016) Olive Jung et al.   Expert Review of Proteomics

Autophagic lysosome reformation dysfunction in glucocerebrosidase deficient cells: relevance to Parkinson disease

(2016) Joana Magalhaes et al.   HUMAN MOLECULAR GENETICS

A New Glucocerebrosidase Chaperone Reduces  -Synuclein and Glycolipid Levels in iPSC-Derived Dopaminergic Neurons from Patients with Gaucher Disease and Parkinsonism

(2016) E. Aflaki et al.   JOURNAL OF NEUROSCIENCE

Activation of  -Glucocerebrosidase Reduces Pathological  -Synuclein and Restores Lysosomal Function in Parkinson's Patient Midbrain Neurons

(2016) J. R. Mazzulli et al.   JOURNAL OF NEUROSCIENCE

A Drosophila Model of Neuronopathic Gaucher Disease Demonstrates Lysosomal-Autophagic Defects and Altered mTOR Signalling and Is Functionally Rescued by Rapamycin

(2016) Kerri J. Kinghorn et al.   JOURNAL OF NEUROSCIENCE

ER Stress and Autophagic Perturbations Lead to Elevated Extracellular α-Synuclein in GBA-N370S Parkinson's iPSC-Derived Dopamine Neurons

(2016) Hugo J.R. Fernandes et al.   Stem Cell Reports

Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study

(2016) Aya Narita et al.   Annals of Clinical and Translational Neurology

Progranulin Recruits HSP70 to β-Glucocerebrosidase and Is Therapeutic Against Gaucher Disease

(2016) Jinlong Jian et al.   EBioMedicine

Association Between Progranulin and Gaucher Disease

(2016) Jinlong Jian et al.   EBioMedicine

Parkinson disease-linked GBA mutation effects reversed by molecular chaperones in human cell and fly models

(2016) Alvaro Sanchez-Martinez et al.   Scientific Reports

Glucocerebrosidase activity in Parkinson’s disease with and withoutGBAmutations

(2015) Roy N. Alcalay et al.   BRAIN

Glucocerebrosidase 1 deficientDanio reriomirror key pathological aspects of human Gaucher disease and provide evidence of early microglial activation preceding alpha-synuclein-independent neuronal cell death

(2015) Marcus Keatinge et al.   HUMAN MOLECULAR GENETICS

Selective loss of glucocerebrosidase activity in sporadic Parkinson’s disease and dementia with Lewy bodies

(2015) Davide Chiasserini et al.   Molecular Neurodegeneration

No evidence for substrate accumulation in Parkinson brains withGBAmutations

(2015) Matthew E. Gegg et al.   MOVEMENT DISORDERS

Glucocerebrosidase gene therapy prevents α-synucleinopathy of midbrain dopamine neurons

(2015) Emily M. Rocha et al.   NEUROBIOLOGY OF DISEASE

Differential effects of severe vs mild GBA mutations on Parkinson disease

(2015) Z. Gan-Or et al.   NEUROLOGY

Reduced glucocerebrosidase is associated with increased α-synuclein in sporadic Parkinson’s disease

(2014) Karen E. Murphy et al.   BRAIN

Ambroxol improves lysosomal biochemistry in glucocerebrosidase mutation-linked Parkinson disease cells

(2014) Alisdair McNeill et al.   BRAIN

The serum lipid profile of Parkinson's disease patients: a study from China

(2014) Xiaoyan Guo et al.   INTERNATIONAL JOURNAL OF NEUROSCIENCE

Mutations in the glucocerebrosidase gene are responsible for Chinese patients with Parkinson’s disease

(2014) Zhe Yu et al.   JOURNAL OF HUMAN GENETICS

GBA-associated Parkinson's disease: Reduced survival and more rapid progression in a prospective longitudinal study

(2014) Kathrin Brockmann et al.   MOVEMENT DISORDERS

A GCase Chaperone Improves Motor Function in a Mouse Model of Synucleinopathy

(2014) Franziska Richter et al.   Neurotherapeutics

iPSC-derived neurons from GBA1-associated Parkinson’s disease patients show autophagic defects and impaired calcium homeostasis

(2014) David C. Schöndorf et al.   Nature Communications

iPSC-Derived Dopamine Neurons Reveal Differences between Monozygotic Twins Discordant for Parkinson’s Disease

(2014) Chris M. Woodard et al.   Cell Reports

Mitochondria and Quality Control Defects in a Mouse Model of Gaucher Disease—Links to Parkinson’s Disease

(2013) Laura D. Osellame et al.   Cell Metabolism

Functional analysis of 11 novel GBA alleles

(2013) Erika Malini et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Altered ceramide acyl chain length and ceramide synthase gene expression in Parkinson's disease

(2013) Sarah K. Abbott et al.   MOVEMENT DISORDERS

Plasma Ceramide and Glucosylceramide Metabolism Is Altered in Sporadic Parkinson's Disease and Associated with Cognitive Impairment: A Pilot Study

(2013) Michelle M. Mielke et al.   PLoS One

Reduced Serum Levels of Triglyceride, Very Low Density Lipoprotein Cholesterol and Apolipoprotein B in Parkinson’s Disease Patients

(2013) Qiang Wei et al.   PLoS One

Histone deacetylase inhibitors increase glucocerebrosidase activity in Gaucher disease by modulation of molecular chaperones

(2013) C. Yang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies

(2013) S. Pablo Sardi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Multicenter Study of Glucocerebrosidase Mutations in Dementia With Lewy Bodies

(2013) Michael A. Nalls et al.   JAMA Neurology

Glucocerebrosidase deficiency in substantia nigra of parkinson disease brains

(2012) Matthew E. Gegg et al.   ANNALS OF NEUROLOGY

Age-specific Parkinson disease risk in GBA mutation carriers: information for genetic counseling

(2012) Huma Q. Rana et al.   GENETICS IN MEDICINE

Discovery, Structure–Activity Relationship, and Biological Evaluation of Noninhibitory Small Molecule Chaperones of Glucocerebrosidase

(2012) Samarjit Patnaik et al.   JOURNAL OF MEDICINAL CHEMISTRY

Glucocerebrosidase Mutations alter the endoplasmic reticulum and lysosomes in Lewy body disease

(2012) Marzena Kurzawa-Akanbi et al.   JOURNAL OF NEUROCHEMISTRY

Endoplasmic Reticulum Stress Is Important for the Manifestations of  -Synucleinopathy In Vivo

(2012) E. Colla et al.   JOURNAL OF NEUROSCIENCE

The link between the GBA gene and parkinsonism

(2012) Ellen Sidransky et al.   LANCET NEUROLOGY

The role of saposin C in Gaucher disease

(2012) Rafael J. Tamargo et al.   MOLECULAR GENETICS AND METABOLISM

Penetrance of Parkinson disease in glucocerebrosidase gene mutation carriers

(2012) M. Anheim et al.   NEUROLOGY

The French Gaucher’s disease registry: clinical characteristics, complications and treatment of 562 patients

(2012) Jérôme Stirnemann et al.   Orphanet Journal of Rare Diseases

High Throughput Screening for Small Molecule Therapy for Gaucher Disease Using Patient Tissue as the Source of Mutant Glucocerebrosidase

(2012) Ehud Goldin et al.   PLoS One

Gaucher Disease Glucocerebrosidase and α-Synuclein Form a Bidirectional Pathogenic Loop in Synucleinopathies

(2011) Joseph R. Mazzulli et al.   CELL

Ex Vivoandin VivoEffects of Isofagomine on Acid β-Glucosidase Variants and Substrate Levels in Gaucher Disease

(2011) Ying Sun et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Is E326K glucocerebrosidase a polymorphic or pathological variant?

(2011) Benjamin Liou et al.   MOLECULAR GENETICS AND METABOLISM

GBA-associated PD presents with nonmotor characteristics

(2011) K. Brockmann et al.   NEUROLOGY

Histone deacetylase inhibitors prevent the degradation and restore the activity of glucocerebrosidase in Gaucher disease

(2011) J. Lu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

CNS expression of glucocerebrosidase corrects  -synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy

(2011) S. P. Sardi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Clinical progression in Parkinson disease and the neurobiology of axons

(2010) Hsiao-Chun Cheng et al.   ANNALS OF NEUROLOGY

Chaperone-Mediated Autophagy Markers in Parkinson Disease Brains

(2010) Lydia Alvarez-Erviti et al.   ARCHIVES OF NEUROLOGY

Characterization of the ERAD process of the L444P mutant glucocerebrosidase variant

(2010) Inna Bendikov-Bar et al.   BLOOD CELLS MOLECULES AND DISEASES

The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of β-glucosidase

(2010) Richie Khanna et al.   FEBS Journal

Induction of the unfolded protein response by α-synuclein in experimental models of Parkinson’s disease

(2010) Arianna Bellucci et al.   JOURNAL OF NEUROCHEMISTRY

Pathogenic Lysosomal Depletion in Parkinson's Disease

(2010) B. Dehay et al.   JOURNAL OF NEUROSCIENCE

Effects of pH and Iminosugar Pharmacological Chaperones on Lysosomal Glycosidase Structure and Stability

(2009) Raquel L. Lieberman et al.   BIOCHEMISTRY

Identification and Characterization of Ambroxol as an Enzyme Enhancement Agent for Gaucher Disease

(2009) Gustavo H. B. Maegawa et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Incidence and remaining lifetime risk of Parkinson disease in advanced age

(2009) J. A. Driver et al.   NEUROLOGY

Multicenter Analysis of Glucocerebrosidase Mutations in Parkinson's Disease

(2009) E. Sidransky et al.   NEW ENGLAND JOURNAL OF MEDICINE

Phenotype, diagnosis, and treatment of Gaucher's disease

(2008) Gregory A Grabowski   LANCET

Total cholesterol and the risk of Parkinson disease

(2008) G. Hu et al.   NEUROLOGY