Rare Disease Film Festival Highlights Patient and Researcher Unity
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 ... Read more
Updates
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Gaucher Patients Want to Be Informed at Diagnosis of Parkinson’s Risk, Study Reports
Most people with Gaucher disease (GD) would like to be informed by their health care provider at diagnosis of their risk of developing Parkinson’s ... Read more -
Ambroxol Plus ERT Is Safe, Could Halt Neurological Progression in Gaucher, Small Study Suggests
Oral treatment with ambroxol — an investigational chaperone therapy — plus enzyme replacement therapy (ERT) was found to be safe over a course of ... Read more -
New Test May Help Select Gaucher Patients Best Suited for Chaperone Therapy, Study Suggests
A laboratory test using blood samples could help identify Gaucher disease (GD) patients likely to respond to experimental chaperone therapies, a study reports. The assay ... Read more -
FDA Grants Orphan Designation to AVR‑RD‑02 Gene Therapy for Gaucher Disease
The U.S. Food and Drug Administration (FDA) granted orphan drug designation to AVR-RD-02, a gene therapy candidate for the treatment of Gaucher disease. AVR‑RD‑02, being ... Read more
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Bone Manifestations Highly Common in Gaucher, Long-term Study Finds
Bone problems are highly common among people with Gaucher disease, with symptoms including chronic ... -
Prevail and Lonza Join Forces to Develop PR001 Gene Therapy for Gaucher Type 2
Prevail Therapeutics and Lonza Pharma & Biotech will collaborate in the development and production ... -
‘Collodion’ Skin Disorder in Newborn Early Sign of Severe Perinatal Type 2 Gaucher, Case Study Suggests
Infants who are born encased in a tight and shiny membrane made of skin ... -
Gaucher Disease Might Be Associated with Rare Types of Blood Cancer, Case Report Suggests
Gaucher disease can predispose patients to rare types of blood cancer, and doctors should ... -
Screening for Multiple Myeloma and Other Blood Cell Disorders May Be Useful with Gaucher, Study Says
Gaucher disease is not common in people with a group of disorders called plasma cell ... -
New Fly Model of Gaucher Disease May Improve Knowledge of Disease Mechanisms, Researchers Say
A new fly model of Gaucher disease may more fully summarize and present the ...
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