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Last Updated: 10/31/2025
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Found 95 publications
Assessing the Value for Money of Enzyme Replacement Therapy in Gaucher Disease Types 1 and 3b: Can Expanded Coverage Be Justified?
Journal: PharmacoEconomics - open
Published: April 08, 2025
Effective encapsulation of therapeutic recombinant enzyme into polymeric nanoparticles as a potential vehicle for lysosomal disease treatment.
Journal: International journal of biological macromolecules
Published: August 14, 2024
Twelve Years of the Gaucher Outcomes Survey (GOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry.
Journal: Journal of clinical medicine
Published: April 19, 2024
A Real-World Investigation of MRI Changes in Bone in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: The EIROS Study.
Journal: Journal of clinical medicine
Published: April 08, 2024
Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry.
Journal: Journal of clinical medicine
Published: March 25, 2024
Safety analysis of self-administered enzyme replacement therapy using data from the Fabry Outcome and Gaucher Outcome Surveys.
Journal: Orphanet journal of rare diseases
Published: March 12, 2024
Evaluation of Lyso-Gb1 as a biomarker for Gaucher disease treatment outcomes using data from the Gaucher Outcome Survey.
Journal: Orphanet journal of rare diseases
Published: February 19, 2024
Impact of COVID-19 pandemic on healthcare delivery for lysosomal storage disorders at a tertiary care public hospital in Mumbai.
Journal: Journal of postgraduate medicine
Published: January 10, 2024
Osteonecrosis in Gaucher disease in the era of multiple therapies: Biomarker set for risk stratification from a tertiary referral center.
Journal: eLife
Published: March 08, 2023
Rapid home therapy infusion of velaglucerase alfa in naïve patients with Gaucher disease.
Journal: Internal medicine journal
Published: February 15, 2023
The Bone Biomarker of Quantitative Chemical Shift Imaging in Patients with Type 1 Gaucher Disease Receiving Low-Dose Long-Term Enzyme Replacement Therapy.
Journal: Journal of clinical medicine
Published: January 05, 2023
Last Updated: 10/31/2025