Elelyso (taliglucerase alfa) provides long-term clinical benefits for people with type 1 Gaucher disease, with a favorable safety profile, according to real-world data from a 10-year study in Albania. The treatment was effective in both patients starting therapy for the first time and…
Sharp Therapeutics said it’s arranging a $10 million private investment to advance the development of ‘901, an oral treatment designed to address the neurological and non-neurological symptoms of Gaucher disease. The financing will come through a non-brokered private placement of common stock. STX Partners, Sharp’s largest shareholder, has…
Measuring levels of glucosylsphingosine (lyso-Gb1), a diagnostic biomarker of Gaucher disease, in children confirmed by newborn screening programs to have the disease may aid a decision about when to start enzyme replacement therapy (ERT). That’s according to a study in Italy that measured lyso-Gb1 levels monthly in three…
