Updates

June 29, 2020June 29, 2020
Arimoclomol Shows Potential to Aid Gaucher Patients in Phase 2 Trial

Arimoclomol, Orphazyme’s investigational therapy for Gaucher disease and other disorders, leads to clinically meaningful, dose-dependent reductions in liver and spleen size in patients with type ... Read more
June 23, 2020June 23, 2020
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization ... Read more
June 22, 2020June 22, 2020
Long-term Cerezyme Followed by Cerdelga Delayed Bone Loss in Gaucher Type 1 Case Study

Using a non-invasive high-resolution imaging method, physicians found that long-term treatment with Cerezyme (imiglucerase) followed by a switch to Cerdelga (eliglustat) seemed to prevent ... Read more
June 15, 2020June 15, 2020
Two Pro-inflammatory Proteins May Be Biomarkers of Gaucher Type 1

People with type 1 Gaucher disease have higher blood levels of two pro-inflammatory proteins than do healthy individuals, a small study suggested. The proteins may ... Read more
June 10, 2020June 10, 2020
Coalition Will Address Racial Disparities in Rare Disease Communities

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting ... 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

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

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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

Potential Targeted Therapy for Cancer Shows Promise for Gaucher Disease, Study Finds

Imaging Technique May Help Monitor Gaucher Severity, Treatment Response, Study Says

Avrobio Pauses Dosing in Phase 1/2 Trial of Gene Therapy for Gaucher

NORD’s Advice on COVID-19: You’re Stronger Than You Think

Common Antibody Abnormalities in Gaucher Not Linked to Disease Severity, Study Finds

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

Cerdelga Leads to Sustained Benefits in Type 1 Gaucher Patients, Study Says

Rare GD1 Genetic Profile Associated With Mild Symptoms, Study Finds

Specific Lipid Traced to Multiple Myeloma Risk in Type 1 Gaucher Patients

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Israeli Gaucher Clinic Treats Arabs With Disease Prevalent Among Ashkenazi Jews

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

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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

Subscribe to our mailing list

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

Potential Targeted Therapy for Cancer Shows Promise for Gaucher Disease, Study Finds

Imaging Technique May Help Monitor Gaucher Severity, Treatment Response, Study Says

Avrobio Pauses Dosing in Phase 1/2 Trial of Gene Therapy for Gaucher

NORD’s Advice on COVID-19: You’re Stronger Than You Think

Common Antibody Abnormalities in Gaucher Not Linked to Disease Severity, Study Finds

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

Cerdelga Leads to Sustained Benefits in Type 1 Gaucher Patients, Study Says

Younger Children With GD1 More Susceptible to Poorer Health-related Quality of Life, Study Suggests

Rare GD1 Genetic Profile Associated With Mild Symptoms, Study Finds

Specific Lipid Traced to Multiple Myeloma Risk in Type 1 Gaucher Patients

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

Israeli Gaucher Clinic Treats Arabs With Disease Prevalent Among Ashkenazi Jews

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