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…
When you live with a rare disorder and other chronic conditions, as I do, there’s a certain type of exhaustion that comes from constantly going from doctor to doctor. It’s not just physical exhaustion, but also mental. I start to feel like my whole life revolves around appointments, tests, and…
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…
