Updates

November 23, 2020November 23, 2020
AVR-RD-02 Gene Therapy Lowers GD Biomarkers in First Patient Dosed in Trial

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 ... Read more
November 18, 2020November 18, 2020
Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

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 ... Read more
November 9, 2020November 10, 2020
UK Mom Knows Fight Far From Over After Losing Baby to Gaucher Type 2

Note: This story was updated Nov. 10, 2020, to clarify that Foggin volunteers with the International Gaucher Alliance and does not hold a formal ... Read more
November 5, 2020November 6, 2020
NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

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 ... Read more
November 2, 2020November 2, 2020
Potential Gene Therapy for Type 2 Gaucher, PR001, Placed on FDA Fast Track

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 ... Read more

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December 14, 2017

ERT Infusion Sessions Could Be Cut from 1 Hour to 10 Minutes, Gaucher Research Shows

News

Reducing infusion time of Vpriv (velaglucerase alfa) enzyme replacement therapy (ERT) in patients with type 1 Gaucher disease from one hour to 10 minutes may be possible using a time-reducing protocol. Developed by a team led by Ari Zimran, ... Read more

Updates

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

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

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

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

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

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

Neuronopathic Gaucher Immune Response Might Protect Brain From Infection

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

Online Awareness Campaigns Aim to Magnetize Supporters Worldwide

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

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

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

Potential SRT for Neuronopathic Gaucher Shows Efficacy in Early Study

Fatty Liver Common, Linked to Scarring, in Type 1 Gaucher, Study Finds

Activities Underway for Newborn Screening Awareness Month

Same But Different Photo Contest Celebrates People With Rare Diseases

Type 3 Gaucher Patients at Greater Risk of Parkinson’s as They Age, Case Series Suggests

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Centogene, Evotec Expand Collaboration to Develop New Gaucher Treatments

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

New Streaming Channel Showcases Rare Disease Films

Measuring Lyso-Gb1 in Dried Blood Spots Can Help Monitor ERT Effectiveness

Children With Gaucher May Need Long-term ERT to Reach Desired Immune Responses, Study Says

25% of Patients in Spain on ERT Report Missed Doses Due to COVID-19

1st Patient Dosed in Trial of AVR-RD-02, Gene Therapy for Gaucher Type 1

Lysogene, Weizmann Institute Team Up on Gene Therapy for Neuronopathic Gaucher

Arimoclomol Shows Potential to Aid Gaucher Patients in Phase 2 Trial

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

Long-term Cerezyme Followed by Cerdelga Delayed Bone Loss in Gaucher Type 1 Case Study

Two Pro-inflammatory Proteins May Be Biomarkers of Gaucher Type 1

Coalition Will Address Racial Disparities in Rare Disease Communities

Cerdelga Effective for Gaucher Type 1 in Real-world Setting, Study Shows

ERT Infusion Sessions Could Be Cut from 1 Hour to 10 Minutes, Gaucher Research Shows

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

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

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

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

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

Subscribe to our mailing list

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

Neuronopathic Gaucher Immune Response Might Protect Brain From Infection

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

Online Awareness Campaigns Aim to Magnetize Supporters Worldwide

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

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

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

Potential SRT for Neuronopathic Gaucher Shows Efficacy in Early Study

Fatty Liver Common, Linked to Scarring, in Type 1 Gaucher, Study Finds

Activities Underway for Newborn Screening Awareness Month

Same But Different Photo Contest Celebrates People With Rare Diseases

Type 3 Gaucher Patients at Greater Risk of Parkinson’s as They Age, Case Series Suggests

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Centogene, Evotec Expand Collaboration to Develop New Gaucher Treatments

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

New Streaming Channel Showcases Rare Disease Films

Measuring Lyso-Gb1 in Dried Blood Spots Can Help Monitor ERT Effectiveness

Children With Gaucher May Need Long-term ERT to Reach Desired Immune Responses, Study Says

25% of Patients in Spain on ERT Report Missed Doses Due to COVID-19

1st Patient Dosed in Trial of AVR-RD-02, Gene Therapy for Gaucher Type 1

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

Lysogene, Weizmann Institute Team Up on Gene Therapy for Neuronopathic Gaucher

Arimoclomol Shows Potential to Aid Gaucher Patients in Phase 2 Trial

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

Long-term Cerezyme Followed by Cerdelga Delayed Bone Loss in Gaucher Type 1 Case Study

Two Pro-inflammatory Proteins May Be Biomarkers of Gaucher Type 1

Coalition Will Address Racial Disparities in Rare Disease Communities

Cerdelga Effective for Gaucher Type 1 in Real-world Setting, Study Shows