Gaucher's disease

(redirected from Gaucher disease type 1)
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Words related to Gaucher's disease

a rare chronic disorder of lipid metabolism of genetic origin

References in periodicals archive ?
Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1.
Cerdelga is reportedly the only first-line oral therapy for certain adult Gaucher disease type 1 patients and Genzyme was selected for bringing an additional treatment option to patients with Gaucher disease, an inherited condition affecting fewer than 10,000 people worldwide.
The company is developing eliglustat tartrate, a capsule taken orally, to provide a convenient treatment alternative for patients with Gaucher disease type 1 and to provide a broader range of treatment options for patients and physicians.
Genzyme disclosed on Tuesday the receipt of approval from the US Food and Drug Administration (FDA) for the only first-line oral therapy for certain adult Gaucher disease type 1 patients, Cerdelga (eliglustat) capsules.
Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), today announced that ENGAGE, the first Phase 3 trial of its investigational oral therapy, eliglustat tartrate, in previously untreated patients with Gaucher disease type 1, met its primary endpoint.
Genzyme reported on Wednesday the receipt of priority review designation for its New Drug Application (NDA) for Cerdelga (eliglustat) for Gaucher disease type 1.
Genzyme was the first company in the world to commercialize a treatment for Gaucher disease, and today Cerezyme[R] (imiglucerase for injection) is the standard of care for patients with Gaucher disease type 1.
Genzyme, a division of Sanofi (Euronext:SAN)(NYSE: SNY), announced today that the US Food and Drug Administration (FDA) has granted a six-month Priority Review designation to its New Drug Application (NDA) for Cerdelga (eliglustat), an investigational oral therapy for adult patients with Gaucher disease type 1.
As part of the agreement, Corden Pharma entered into a supply contract to provide Genzyme with materials needed for the production of eliglustat tartrate, an investigational treatment for Gaucher disease type 1 that is currently in phase 3 clinical trials.
Genzyme said on Friday that it has presented positive new data from its Phase 3 ENGAGE and ENCORE studies of its investigational oral therapy eliglustat tartrate for Gaucher disease type 1 at the 9th Annual Lysosomal Disease Network WORLD Symposium in Orlando, Fla.
Genzyme Corporation (NASDAQ: GENZ) today announced three-year follow-up data from patients enrolled in the phase 2 clinical trial for its investigational oral therapy for Gaucher disease type 1 known as eliglustat tartrate.
Additionally, ICIG will execute a five-year supply contract to provide Genzyme with materials needed for the production of eliglustat tartrate, an investigational treatment for Gaucher disease Type 1 that is currently in phase 3 clinical trials.
Eliglustat tartrate, a capsule taken orally, is being developed to provide a treatment alternative for adult patients with Gaucher disease type 1, and to offer a broader range of treatment options for patients and physicians to achieve individual therapeutic goals.
As part of the agreement, ICIG will enter into a five-year supply contract to provide Genzyme with materials needed for the production of eliglustat tartrate, an investigational treatment for Gaucher disease Type 1 that is currently in phase 3 clinical trials.
The second trial, ENGAGE, is a randomized, double-blind, placebo-controlled study for patients with a confirmed diagnosis of Gaucher disease type 1.