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January 18, 2021January 18, 2021

Cerdelga Seen To Safely, Effectively Treat GD1 After Long-term ERT Use

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Cerdelga (eliglustat) safely and effectively treated three adults with Gaucher disease type 1 (GD1), who switched from long-term use of enzyme replacement therapy ... Read more

January 12, 2021January 12, 2021

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

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The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at ... Read more

January 11, 2021January 11, 2021

Gene Therapy, Now in GD1 Trial, Shows Efficacy in Mice at Varying Disease Stages

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An investigational gene therapy aiming to restore GBA gene function — which is defective in people with Gaucher disease — showed ... Read more

January 4, 2021January 4, 2021

Gaucher Patients Not at Increase Risk From COVID-19, Study Says

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People with Gaucher disease are not at increased risk of severe disease if they become infected with SARS-CoV-2, the virus that ... Read more

December 21, 2020December 21, 2020

Lilly Acquires Prevail and Gene Therapy for Neuronopathic Gaucher

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Eli Lilly reached an agreement to acquire Prevail Therapeutics, including its clinical program for PR001, an experimental one-time gene therapy for neuronopathic Gaucher ... Read more

December 14, 2020December 14, 2020

Stem Cell Transplant Treats Lung Involvement in Young Patient, Study Reports

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A bone marrow transplant successfully reduced the severe lung involvement that developed in a 5-year-old boy with Gaucher disease who was ... Read more

December 7, 2020December 7, 2020

Prevail’s Gene Therapy PR001 Granted US Patent for Neuronopathic Gaucher

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The U.S. Patent and Trademark Office has issued a patent related to PR001, Prevail Therapeutics‘ investigational gene therapy for neuronopathic Gaucher ... Read more

December 1, 2020December 1, 2020