Gene Therapy for Gaucher Disease: AAV, Lentivirus, & More
Gaucher disease occurs when people have mutations, or changes, in... Read more
Gaucher disease occurs when people have mutations, or changes, in... Read more
Two thirds of Gaucher patients have some evidence of bone... Read more
Rare diseases can affect all aspects of a patient’s life,... Read more
In our 2019 Impact Report, we take a look at... Read more
AVROBIO announced that the U.S. Food and Drug Administration (FDA)... Read more
Enzyme replacement therapy (ERT) was first approved for use by... Read more
Growth in Gaucher: Managing Bone Health Dr. Heather A. Lau,... Read more
Illuminating Lyso-Gyl 1: Does the Key Biomarker of Gaucher Disease... Read more
Medical research has always been important for furthering our understanding... Read more
U.S. Food and Drug Administration (FDA) has accepted Prevail’s Investigational... Read more
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