Search Results for: gaucher disease type 1

February 1, 2021February 2, 2021

Newly Found RNA Molecules May Be Biomarkers for Disease Progression

News

A set of small molecules called microRNA (miRNA), found in a recent study, show potential both as biomarkers and as therapeutic targets of Gaucher disease. Determining differences in the levels ... Read more

January 25, 2021January 25, 2021

Cerezyme Effective Over 20 Years in Easing GD1 Symptoms, Data Show

News

When given over two decades, Cerezyme (imiglucerase) was effective at easing most symptoms of Gaucher disease type 1 (GD1), according to long-term data from an international patient registry. Importantly, these ... Read more

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 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 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

News

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

News

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

PR001

PR001 is an experimental gene therapy for neuronopathic Gaucher disease, primarily type 2 but also type 3 disease. Prevail Therapeutics — recently acquired by Eli Lilly — oversaw initial development of ... Read more

December 7, 2020December 7, 2020

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

News

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

November 9, 2020November 10, 2020

UK Mom Knows Fight Far From Over After Losing Baby to Gaucher Type 2

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Note: This story was updated Nov. 10, 2020, to clarify that Foggin volunteers with the International Gaucher Alliance and does not hold a formal position with Prevail. In addition, the ... Read more

November 2, 2020November 2, 2020

Potential Gene Therapy for Type 2 Gaucher, PR001, Placed on FDA Fast Track

News

The U.S. Food and Drug Administration (FDA) has given fast track designation to PR001, Prevail Therapeutics‘ investigational gene therapy for neuronopathic Gaucher disease. The FDA previously granted the therapy — now in an ... Read more