Safety of measles, rubella and mumps vaccines in adults: a prospective cohort study.

Background: In recent years, multiple outbreaks of measles associated with vaccine hesitancy occurred in high-income countries, where measles incidence had previously been low. Most safety data about the measles, mumps and rubella (MMR) vaccine are derived from studies conducted among children, whereas evidence regarding the safety profile of the vaccine in adults is scarce.

Methods: In 2017, during an outbreak of measles in Europe, Israeli travellers to high-risk locations who were incompletely vaccinated, were urged to complete the two MMR vaccination schedule before their travel.

Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience.

Enzyme replacement therapy is the standard of care for symptomatic Gaucher disease. Velaglucerase alfa is a human beta-glucocerebrosidase produced in a well-characterized human cell line. A 9-month phase 1/2 open-label, single-center trial and ongoing extension study were conducted to evaluate safety and efficacy of velaglucerase alfa.

Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.

Enzyme replacement therapy (ERT) with imiglucerase reduces hepatosplenomegaly and improves hematologic parameters in Gaucher disease type 1 within 6-24 months. Miglustat reduces organomegaly, improves hematologic parameters, and reverses bone marrow infiltration. This trial evaluates miglustat in patients clinically stable on ERT.

Automated system to detect low-grade underlying inflammatory profile: Gaucher disease as a model.

Patients with Gaucher disease, perhaps due to chronic storage of glycolipids, apparently harbor a subclinical or underlying inflammation. Quantification of a baseline inflammatory profile in patients with Gaucher disease is more impressive when compared with that of matched healthy controls in a systematic, automated fashion. A mean of 16 healthy controls was generated for each of 50 patients with Gaucher disease by applying variables relating to potential for inflammatory features, for example, atherothrombotic risk factors.