News

January 18, 2021January 18, 2021

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

News

Cerdelga (eliglustat) safely and effectively treated three adults with Gaucher disease type 1 (GD1), who switched from long-term use of enzyme replacement therapy (ERT) to this substrate reduction therapy (SRT), a study ... Read more

January 12, 2021January 12, 2021

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

News, syndicated

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient ... Read more

January 11, 2021January 11, 2021

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

News

An investigational gene therapy aiming to restore GBA gene function — which is defective in people with Gaucher disease — showed efficacy both before and after symptom development in a ... 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 causes COVID-19, new research indicates. The results were published ... 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 disease. Prevail has a portfolio that includes early and ... 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 receiving enzyme replacement therapy (ERT), a study reported. The ... 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 disease. The composition of matter patent grants coverage until ... Read more

December 1, 2020December 1, 2020

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

News, syndicated

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) ... Read more

November 26, 2020November 26, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

News, syndicated

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a ... Read more

November 23, 2020November 23, 2020

AVR-RD-02 Gene Therapy Lowers GD Biomarkers in First Patient Dosed in Trial

News

A single infusion of AVR-RD-02, Avrobio’s experimental gene therapy for Gaucher disease type 1, was able to lower the levels of two disease biomarkers within three months in the first ... Read more