Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening ... Read more
Updates
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Cough Medicine Ambroxol Relieves Chronic Bone Pain in Woman With Gaucher, Report Says
The cough medicine ambroxol may be effective for relieving persistent bone pain in people with Gaucher disease, a case report suggests. The report describes how ... Read more -
Fat ‘Floaters’ Impairing Vision in GD3 Patient Highlight Need for Monitoring, New Therapies, Case Study Reports
A young man with Gaucher disease type 3 (GD3) developed fat deposits in the eye, called floaters, that impaired his vision despite long-term treatment ... Read more -
FDA Grants Orphan Drug, Rare Pediatric Disease Designations to PR001 Investigational Gene Therapy
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Prevail Therapeutics‘ investigational therapy PR001 for the treatment of Gaucher disease. ... Read more
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Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day ... -
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 ... -
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan ... -
IGA, Kantar Partner to Build Global Gaucher Registry for Type 2 and 3 Patients
The International Gaucher Alliance (IGA), which includes members from 54 countries, has announced a partnership ... -
Gaucher Mouse Model Mimics Broad Set of Symptoms to Aid in Therapy Discovery, Study Says
An in-depth characterization of a mouse model of Gaucher disease shows that it effectively ...
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