European Initiative Targets Diagnosis, Treatment of Rare Diseases
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while ... Read more
Updates
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New Mouse Model of Gaucher Type 3 May Aid Studies into Disease and Possible Treatments
A new mouse model of Gaucher disease type 3 may be more useful for studies of disease biology and preliminary tests of possible treatments ... Read more -
Lyso-Gb1 Biomarker May Be Useful for Monitoring Disease Activity in Children, Study Suggests
Glucosylsphingosine (lyso-Gb1) seems to be a useful biomarker for monitoring disease activity in children with Gaucher disease, helping physicians determine when enzyme replacement therapy ... Read more -
Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients
The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have ... Read more -
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing ... Read more
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Patients on Enzyme Replacement Therapy May Be Prone to Psychiatric Disorders, Study Suggests
Gaucher disease patients on enzyme replacement therapy may be prone to psychiatric disorders, according ... -
NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy ... -
Cerdelga Has Favorable Long-term Safety Profile in Patients with Type 1 Gaucher Disease, Pooled Trial Data Show
Cerdelga (eliglustat) has a favorable long-term safety profile consistent with previous observations among patients ... -
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to ... -
Non-motor Symptoms Preceding Parkinson’s Onset Prevalent in Gaucher Type 1, Study Finds
Non-motor symptoms associated with the eventual development of Parkinson’s disease are prevalent in 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