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April 6, 2020April 6, 2020

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

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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 a Phase 1/2 study testing ... Read more

March 30, 2020March 30, 2020

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

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Although common in patients with Gaucher disease, antibody abnormalities are not associated with disease severity, a large French study reports. Still, monitoring these patients is important because they may ... Read more

March 26, 2020March 26, 2020

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

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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 of the coronavirus disease COVID-19 pandemic. ... Read more

March 25, 2020March 25, 2020

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

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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 nonprofit she chairs will see ... Read more

March 23, 2020March 23, 2020

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

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Long-term treatment with Cerdelga (eliglustat) safely and consistently lessens or stabilizes disease severity in adults with Gaucher disease type 1 (GD1), according to a six-year study in five patients. The findings ... Read more

March 19, 2020March 19, 2020

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

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Children with type 1 Gaucher disease are more likely to experience social withdrawal, anxiety, and depression with poorer health-related quality of life than their peers without the disorder, researchers ... Read more

March 16, 2020March 16, 2020

Rare GD1 Genetic Profile Associated With Mild Symptoms, Study Finds

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Children and adults with a rare genetic profile causing type 1 Gaucher disease (GD1) may exhibit mild symptoms and not require treatment, research suggests. The study, “The N370S/R496H genotype ... Read more

March 12, 2020March 12, 2020

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

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Developing abnormal antibodies specific to glucosylsphingosine — a fat that builds up in cells of Gaucher disease patients — may be the cause of monoclonal gammopathy, and explain why patients ... Read more

March 9, 2020March 9, 2020

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

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The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more

March 5, 2020March 5, 2020

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

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In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the ... Read more

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