Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one ... Read more
Updates
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New GBA Mutation May Be Linked to Perinatal Lethal Gaucher Disease, Case Report Suggests
A new mutation, H413P, that may be associated with severe symptoms was discovered in a Chinese baby with perinatal lethal Gaucher disease, a case ... Read more -
Gain Therapeutics and Partners Win €1.4M Grant to Develop Treatments for Gaucher and Other Diseases
A €1.4 million grant from the Eurostars-2 program, with funding from others, will support Gain Therapeutics and its partners in developing treatment candidates for Gaucher ... Read more -
Ways Needed to Ease High Cost of Gaucher Care to Patients and State in Iran, Study Says
Gaucher disease is among the more costly of chronic diseases to treat in Iran, and efforts are needed to minimize the financial burden facing ... Read more -
Psychologist Living with Gaucher Strives to Be ‘Role Model of Success’
When school psychologist Wayne Rosenfield was 5 years old, he had an enlarged spleen. Doctors in his hometown of Springfield, Massachusetts, brushed it off, ... Read more
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Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of ... -
Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, ... -
Scoring Swallowing Problems Helps Stage Neurological Decline in Type 2 GD Infants
A scoring system of swallowing difficulties may be a simple and valuable way of ... -
Scientist Gets Funding to Create 3-D Bone Tissue Model for Gaucher
A Penn State University researcher will use a $65,000 grant from the Lysosomal and Rare ... -
Centogene Rolls Out Free Biomarker App for Gaucher Patients
Germany’s Centogene is introducing a free smartphone app that lets people with Gaucher disease ... -
Pentosan Polysulfate May Reduce Inflammation in Gaucher, Fabry, Study Suggests
Pentosan polysulfate (PPS), an approved medicine, may be a potential add-on treatment for Gaucher ...
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