Updates

February 22, 2021February 22, 2021
Blood Test Plus Algorithm Might Help to More Easily Spot Gaucher Type 1

An enlarged spleen or low platelets counts — or both — in a person with difficult-to-diagnose complaints may be a sign of type 1 Gaucher ... Read more
February 18, 2021February 18, 2021
Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by ... Read more
February 15, 2021February 15, 2021
AVR-RD-02 Gene Therapy Still Showing Efficacy in 1st Treated Patient in Trial

Updated, six-month data from a Phase 1/2 trial show a continued drop in the levels of two disease biomarkers in the first patient given a ... Read more
February 10, 2021February 11, 2021
Pandemic Won’t Stop Rare Disease Day on Feb. 28

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness ... Read more
February 8, 2021February 8, 2021
FDA Grants Orphan Drug Status to M6P Gene Therapy

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to two gene therapies that M6P Therapeutics is developing — one intended for ... 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, MD, ... Read more

Updates

Blood Test Plus Algorithm Might Help to More Easily Spot Gaucher Type 1

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

AVR-RD-02 Gene Therapy Still Showing Efficacy in 1st Treated Patient in Trial

Pandemic Won’t Stop Rare Disease Day on Feb. 28

FDA Grants Orphan Drug Status to M6P Gene Therapy

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NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

Newly Found RNA Molecules May Be Biomarkers for Disease Progression

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Cerezyme Effective Over 20 Years in Easing GD1 Symptoms, Data Show

NORD’s Caregiver Respite Program Continues Through Pandemic

Cerdelga Seen To Safely, Effectively Treat GD1 After Long-term ERT Use

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

Gene Therapy, Now in GD1 Trial, Shows Efficacy in Mice at Varying Disease Stages

Gaucher Patients Not at Increase Risk From COVID-19, Study Says

Lilly Acquires Prevail and Gene Therapy for Neuronopathic Gaucher

Stem Cell Transplant Treats Lung Involvement in Young Patient, Study Reports

Prevail’s Gene Therapy PR001 Granted US Patent for Neuronopathic Gaucher

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

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

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

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