Prevail Therapeutics and Lonza Pharma & Biotech will collaborate in the development and production of Prevail’s PR001…
Articles by Alice Melao
Infants who are born encased in a tight and shiny membrane made of skin cells that resembles plastic wrap, and…
Gaucher disease is not common in people with a group of disorders called plasma cell dyscrasias (PCDs), which include…
Treatment with Cerezyme (imiglucerase), Sanofi Genzyme‘s enzyme replacement therapy, can effectively reverse severe pulmonary and liver complications…
Patients with Gaucher disease type 1 may experience a complication involving the lower portion of the spinal cord, known as…
Elelyso (taliglucerase alfa) is safe for use by breastfeeding women with Gaucher disease, not representing a risk to the…
Early and accurate diagnosis of Gaucher disease can prevent poor outcome in children and adolescents affected by the rare illness,…
A newly identified mutation in the GBA gene causes errors in gene processing that trigger the development of Gaucher disease…
AVROBIO is advancing its gene therapy for Gaucher disease, AVR-RD-02, into a Phase 1/2 clinical trial after Health Canada raised no…
A new mutation on the DNA coding sequence of the beta-glucocerebrosidase enzyme was identified and linked to Gaucher disease…