Gaucher Disease Type 1 Latest Advances

Find the Latest Research About Gaucher Disease Type 1

Last Updated: 06/30/2026

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Found 396 publications

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

Journal: Zhonghua yi xue za zhi

Published: May 20, 2026

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.

Journal: Neurology

Published: May 05, 2026

persistent pain and fatigue drive reduced quality of life in treated Gaucher disease type 1: a cross-sectional analysis.

persistent pain and fatigue drive reduced quality of life in treated Gaucher disease type 1: a cross-sectional analysis.

Journal: Orphanet journal of rare diseases

Published: April 09, 2026

Left Ventricular Global Longitudinal Strain as an Early Predictor of Disease Severity Progression in Gaucher Disease Type 1.

Left Ventricular Global Longitudinal Strain as an Early Predictor of Disease Severity Progression in Gaucher Disease Type 1.

Journal: Echocardiography (Mount Kisco, N.Y.)

Published: March 12, 2026

A 53-week, open-label phase IIIb study of velaglucerase alfa in Chinese patients with type 1 Gaucher disease: Safety, efficacy, and pharmacokinetics.

A 53-week, open-label phase IIIb study of velaglucerase alfa in Chinese patients with type 1 Gaucher disease: Safety, efficacy, and pharmacokinetics.

Journal: Molecular genetics and metabolism reports

Published: March 02, 2026

Increased intervals in enzyme replacement therapy for stable type 1 Gaucher disease: A non-inferiority sequential trial emulation.

Increased intervals in enzyme replacement therapy for stable type 1 Gaucher disease: A non-inferiority sequential trial emulation.

Journal: Journal of internal medicine

Published: February 28, 2026

Transcriptomic Profiling of Monozygotic Twins with Type 1 Gaucher Disease.

Transcriptomic Profiling of Monozygotic Twins with Type 1 Gaucher Disease.

Journal: Life (Basel, Switzerland)

Published: February 18, 2026

Improvement of Bone Mineral Density in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: Results from Clinical Studies.

Improvement of Bone Mineral Density in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: Results from Clinical Studies.

Journal: Journal of clinical medicine

Published: January 19, 2026

Real-World Effectiveness and Safety of Eliglustat in Adult Patients with Gaucher Disease Type 1: A Multicenter Retrospective Study in China.

Real-World Effectiveness and Safety of Eliglustat in Adult Patients with Gaucher Disease Type 1: A Multicenter Retrospective Study in China.

Journal: Journal of clinical medicine

Published: January 15, 2026

Genomic Structural Equation Modeling Provides an Initial View of the Genetic Architecture Related to Type 1 Gaucher Disease.

Genomic Structural Equation Modeling Provides an Initial View of the Genetic Architecture Related to Type 1 Gaucher Disease.

Journal: Human mutation

Published: December 27, 2025

Homozygous GBA1 p.T82I variant in type 1 Gaucher disease: clinical and biochemical characterization.

Homozygous GBA1 p.T82I variant in type 1 Gaucher disease: clinical and biochemical characterization.

Journal: Personalized medicine

Published: December 24, 2025

Rare in Rare: Overlapping Clinical Features in a Patient With Both Gaucher Disease Type 1 and B4GALT-CDG: Expanding the Clinical Spectrum With a Novel Pathogenic Variant.

Rare in Rare: Overlapping Clinical Features in a Patient With Both Gaucher Disease Type 1 and B4GALT-CDG: Expanding the Clinical Spectrum With a Novel Pathogenic Variant.

Journal: Clinical genetics

Published: October 03, 2025

Showing 1-12 of 396

Last Updated: 06/30/2026