Avrobio Pauses Dosing in Phase 1/2 Trial of Gene Therapy for Gaucher
Due to the current COVID-19 outbreak, Avrobio announced that it has paused dosing new patients in clinical trials of its gene therapy candidates, including ... Read more
Updates
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NORD’s Advice on COVID-19: You’re Stronger Than You Think
First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to ... Read more -
Common Antibody Abnormalities in Gaucher Not Linked to Disease Severity, Study Finds
Although common in patients with Gaucher disease, antibody abnormalities are not associated with disease severity, a large French study reports. Still, monitoring these patients ... Read more -
NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because ... Read more -
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based ... Read more
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Cerdelga Leads to Sustained Benefits in Type 1 Gaucher Patients, Study Says
Long-term treatment with Cerdelga (eliglustat) safely and consistently lessens or stabilizes disease severity in adults ... -
Rare GD1 Genetic Profile Associated With Mild Symptoms, Study Finds
Children and adults with a rare genetic profile causing type 1 Gaucher disease (GD1) ... -
Specific Lipid Traced to Multiple Myeloma Risk in Type 1 Gaucher Patients
Developing abnormal antibodies specific to glucosylsphingosine — a fat that builds up in cells ... -
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
The number of treatments for children with rare diseases has grown over the past decade, ... -
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media ...
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