Gaucher Disease Latest Advances

Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies.

Journal: eLife

Published: June 23, 2026

Diagnosis and follow-up of the first case of Gaucher disease under enzyme replacement therapy in Senegal.

Journal: Ghana medical journal

Published: June 05, 2026

Cumulative Antigen Suppression Reduces Clonal Plasma Cell Evolution in Gaucher Disease.

Journal: American journal of hematology

Published: May 27, 2026

Fetal/Perinatal Lethal Gaucher Disease: A Case Series.

Journal: Pediatric and developmental pathology : the official journal of the Society for Pediatric Pathology and the Paediatric Pathology Society

Published: May 21, 2026

Efficacy and safety of eliglustat tartrate in adults with Gaucher disease type 1

Journal: Zhonghua yi xue za zhi

Published: May 20, 2026

Pharmacotherapies for Gaucher disease: What's New on the horizon?

Journal: Expert opinion on pharmacotherapy

Published: May 19, 2026

Correction to "Evaluation of Induced Pluripotent Stem Cell-Derived Dopaminergic Neurons from Siblings with Gaucher Disease Discordant for Parkinsonism".

Journal: Movement disorders : official journal of the Movement Disorder Society

Published: May 18, 2026

Emerging pharmacotherapies in Gaucher disease.

Journal: Expert opinion on emerging drugs

Published: May 14, 2026

Age-Specific Parkinson Disease Risk in Gaucher Disease Type 1: Data From the ICGG Gaucher Registry.

Journal: Neurology

Published: May 05, 2026

Risk Is Not Destiny: Unmasked Penetrance of Parkinson Disease in Gaucher Disease.

Journal: Neurology

Published: May 05, 2026

A Multiscale Signaling-Biophysical Framework Reveals Mechanisms of Macrophage-Mediated RBC Clearance in Sickle Cell and Gaucher Disease.

Journal: bioRxiv : the preprint server for biology

Published: May 04, 2026

From Lysosomal Storage to Neurodegeneration: Sphingolipid Signaling as a Driver of CNS Pathology and Biomarker Strategy in Neuronopathic Gaucher Disease.

Journal: International journal of molecular sciences

Published: April 29, 2026

Gaucher Disease Latest Advances

Find the Latest Research About Gaucher Disease

Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies.

Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies.

Diagnosis and follow-up of the first case of Gaucher disease under enzyme replacement therapy in Senegal.

Diagnosis and follow-up of the first case of Gaucher disease under enzyme replacement therapy in Senegal.

Cumulative Antigen Suppression Reduces Clonal Plasma Cell Evolution in Gaucher Disease.

Cumulative Antigen Suppression Reduces Clonal Plasma Cell Evolution in Gaucher Disease.

Fetal/Perinatal Lethal Gaucher Disease: A Case Series.

Fetal/Perinatal Lethal Gaucher Disease: A Case Series.

Efficacy and safety of eliglustat tartrate in adults with Gaucher disease type 1

Efficacy and safety of eliglustat tartrate in adults with Gaucher disease type 1

Pharmacotherapies for Gaucher disease: What's New on the horizon?

Pharmacotherapies for Gaucher disease: What's New on the horizon?

Correction to "Evaluation of Induced Pluripotent Stem Cell-Derived Dopaminergic Neurons from Siblings with Gaucher Disease Discordant for Parkinsonism".

Correction to "Evaluation of Induced Pluripotent Stem Cell-Derived Dopaminergic Neurons from Siblings with Gaucher Disease Discordant for Parkinsonism".

Emerging pharmacotherapies in Gaucher disease.

Emerging pharmacotherapies in Gaucher disease.

Age-Specific Parkinson Disease Risk in Gaucher Disease Type 1: Data From the ICGG Gaucher Registry.

Age-Specific Parkinson Disease Risk in Gaucher Disease Type 1: Data From the ICGG Gaucher Registry.

Risk Is Not Destiny: Unmasked Penetrance of Parkinson Disease in Gaucher Disease.

Risk Is Not Destiny: Unmasked Penetrance of Parkinson Disease in Gaucher Disease.

A Multiscale Signaling-Biophysical Framework Reveals Mechanisms of Macrophage-Mediated RBC Clearance in Sickle Cell and Gaucher Disease.

A Multiscale Signaling-Biophysical Framework Reveals Mechanisms of Macrophage-Mediated RBC Clearance in Sickle Cell and Gaucher Disease.

From Lysosomal Storage to Neurodegeneration: Sphingolipid Signaling as a Driver of CNS Pathology and Biomarker Strategy in Neuronopathic Gaucher Disease.

From Lysosomal Storage to Neurodegeneration: Sphingolipid Signaling as a Driver of CNS Pathology and Biomarker Strategy in Neuronopathic Gaucher Disease.