News

November 18, 2020November 18, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about ... 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, ... Read more

November 5, 2020November 6, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

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The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit ... Read more

November 2, 2020November 2, 2020

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

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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 ... Read more

October 29, 2020October 29, 2020

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

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A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar ... Read more

October 26, 2020October 26, 2020

Neuronopathic Gaucher Immune Response Might Protect Brain From Infection

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Neuronopathic Gaucher disease (nGD) induces an immune response that protects against viral infections in the brain, according to an early study in animals. Understanding how this happens and whether ... Read more

October 21, 2020October 21, 2020

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

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A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a ... Read more

October 19, 2020October 19, 2020

Online Awareness Campaigns Aim to Magnetize Supporters Worldwide

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From tweeting disease facts to taking photos with licence plates, supporters worldwide are participating in International Gaucher Disease Awareness Month, set aside each October to bring global attention to ... Read more

October 12, 2020October 12, 2020

Gaucher Type 1 Therapy AVR-RD-02 Granted EU Orphan Drug Status

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An experimental gene therapy known as AVR-RD-02 has received orphan drug designation from the European Commission for the treatment of Gaucher disease type 1. In the European Union, orphan drug ... Read more

October 6, 2020October 6, 2020

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

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Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare ... Read more