Updates

May 28, 2020May 28, 2020
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays ... Read more
May 25, 2020May 25, 2020
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 ... Read more
May 21, 2020May 21, 2020
Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, ... Read more
May 20, 2020
Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together ... Read more
May 19, 2020May 19, 2020
Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together ... 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

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

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

Subscribe to our mailing list

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

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

Blood Levels of CCL18 Can Assess Gaucher Severity in Clinical Practice, Study Suggests

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

Cough Medicine Ambroxol Relieves Chronic Bone Pain in Woman With Gaucher, Report Says

Fat ‘Floaters’ Impairing Vision in GD3 Patient Highlight Need for Monitoring, New Therapies, Case Study Reports

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

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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

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

Subscribe to our mailing list

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

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

Blood Levels of CCL18 Can Assess Gaucher Severity in Clinical Practice, Study Suggests

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

Cough Medicine Ambroxol Relieves Chronic Bone Pain in Woman With Gaucher, Report Says

Fat ‘Floaters’ Impairing Vision in GD3 Patient Highlight Need for Monitoring, New Therapies, Case Study Reports

Pin It on Pinterest