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…
