Updates

September 14, 2020September 14, 2020
Fatty Liver Common, Linked to Scarring, in Type 1 Gaucher, Study Finds

Liver steatosis, a condition characterized by the buildup of fatty tissue inside the liver, is common in adults with type 1 Gaucher disease and is ... Read more
September 10, 2020September 10, 2020
Activities Underway for Newborn Screening Awareness Month

As it does each September during Newborn Screening Awareness Month, Baby’s First Test is sharing information and stories that highlight efforts throughout the U.S. to ... Read more
September 3, 2020September 3, 2020
Same But Different Photo Contest Celebrates People With Rare Diseases

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for ... Read more
August 31, 2020August 31, 2020
Type 3 Gaucher Patients at Greater Risk of Parkinson’s as They Age, Case Series Suggests

Much like people with type 1 Gaucher disease (GD), patients with type 3 may be at greater risk of developing features of Parkinson’s disease as ... Read more
August 25, 2020August 25, 2020
Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare ... Read more

Subscribe to our mailing list

Get regular updates delivered to your email box.

Thanks! Please check your email for a confirmation email.

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

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

Subscribe to our mailing list

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

Liver Health Needs Constant Monitoring in Gaucher Patients, Study Says

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

RANK Pathway Components May Be Markers of Bone Disease in Gaucher, Study Says

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Cognitive Impairment, Depression May Predict Parkinsonism in Gaucher Patients, Study Says

GCase Screening in Dried Blood Spots Can Diagnose GD Early in High-risk Groups, Study Says

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

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

Pin It on Pinterest

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

Subscribe to our mailing list

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

Liver Health Needs Constant Monitoring in Gaucher Patients, Study Says

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

RANK Pathway Components May Be Markers of Bone Disease in Gaucher, Study Says

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Cognitive Impairment, Depression May Predict Parkinsonism in Gaucher Patients, Study Says

GCase Screening in Dried Blood Spots Can Diagnose GD Early in High-risk Groups, Study Says

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Pin It on Pinterest