People with Gaucher disease type 1 are known to be at increased risk of Parkinson’s disease, but data from a new study show that fewer than 15% of those with this disease type — marked by an absence of neurological symptoms — will develop Parkinson’s in their lifetime.
News
The U.S. Food and Drug Administration (FDA) has granted priority review to an application for venglustat, an experimental substrate reduction therapy (SRT) that Sanofi is developing for Gaucher disease type 3. The FDA grants priority review to applications for therapies that may offer significant improvements for serious…
Extending genetic testing beyond the person initially diagnosed with Gaucher disease can uncover additional affected relatives who might otherwise go undetected, according to a new study. When researchers tested family members of Gaucher patients, some relatives, including siblings, children, grandparents, aunts, and uncles, were either genetic carriers or undiagnosed…
One-time treatment with avigbagene parvec, a gene therapy being developed by Spur Therapeutics, may improve bone health in adults with Gaucher disease. That’s according to data from a now-completed Phase 1/2 clinical trial, GALILEO-1 (NCT05324943), and its ongoing long-term extension study, GALILEO-2 (NCT06545136), showing that…
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…
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