News

Colchicine, an approved anti-inflammatory medication for treating gout, significantly reduced fat accumulation and signs of cellular damage in a cell model of Gaucher disease, according to a new study. The lab findings suggest that the oral treatment — which was identified for potential use in Gaucher with the aid…

VPRIV (velaglucerase alfa) may help improve bone marrow health in adults with Gaucher disease type 1, but its benefit for bone strength appears to be modest and may vary from patient to patient, a small Phase 4 clinical study found. The study, “Improvement of Bone Mineral Density…

Long-term treatment with Cerezyme (imiglucerase), or its discontinued predecessor Ceredase (alglucerase), led to rapid and progressive improvements in blood counts, growth, and bone health, as well as reductions in liver and spleen size, in children with Gaucher disease (GD) — including among youngsters with a more severe disease…

Researchers have developed a therapy using messenger RNA (mRNA) that has the potential to restore production of the enzyme missing in people with Gaucher disease, according to a mouse study. Messenger RNA is the template molecule that carries genetic instructions for protein production. The therapy, packaged in tiny fatty…

Monoclonal gammopathies, benign blood conditions that can progress to certain blood cancers such as myeloma, are 10 times more common in people with Gaucher disease than in the general population, a study found. Researchers screened more than 500 patients and found that more than 30% of adults with…

For children and adults with Gaucher disease type 1 who are clinically stable on enzyme replacement treatment (ERT), spacing treatment to every three to four weeks appears to work as well as the usual every-two-week schedule, according to a study using data from France. Because spacing treatment also…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy status to Sanofi‘s experimental oral medication for Gaucher disease type 3, venglustat, which has shown potential in a clinical trial for improving measures of neurological function in people with this form of the genetic disorder. While there…

Treatment with VPRIV (velaglucerase alfa) was generally safe and showed benefits — including stable blood levels — among children and adolescents with Gaucher disease type 1, a new review of published studies has found. Evidence also suggests the approved enzyme replacement therapy (ERT) may help manage nonneurological…

Treatment with Sanofi’s enzyme replacement therapy (ERT) Cerezyme (imiglucerase) during pregnancy was associated with favorable outcomes for women with type 1 Gaucher disease and their babies, according to data from an international patient registry. Among more than 100 pregnancies, most resulted in live births, and most infants…

Transient elastography, a noninvasive test that uses ultrasound to detect liver stiffness and fat, may be used to assess tissue scarring and excess fat in the liver of adults with type 1 Gaucher disease, a study showed. More severe liver scarring, or fibrosis, was significantly associated with higher blood…