News

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…

Gaucher disease may alter how macrophages, key immune cells involved in the disease, produce energy and process fats, according to a new study using computational modeling. Based on their model, researchers identified a few genes that may help regulate these metabolic changes in Gaucher cells. The scientists called for…

Avigbagene parvec, a one-time gene therapy being developed by Spur Therapeutics for Gaucher disease type 1, helped four of six adults in a Phase 1/2 study remain off their standard treatment for up to about two years, trial data show. While stopping treatment typically leads to a rapid…

Sanofi‘s experimental oral therapy venglustat was better than its enzyme replacement therapy (ERT) Cerezyme (imiglucerase) at easing neurological symptoms in people with Gaucher disease type 3. That’s according to new data from the LEAP2MONO Phase 3 clinical trial (NCT05222906), presented as late-breaking research last week…