Treatment with Lumizyme (alglucosidase alfa) improved lung function in the short term and slowed lung function decline over the long term among people with late-onset Pompe disease (LOPD), according to a 13-year, real-world analysis. Patients with poor lung function before treatment saw the greatest early gains, while those…
Lumizyme (alglucosidase alfa)
People with late-onset Pompe disease (LOPD) treated with Nexviazyme (avalglucosidase alfa) may be more than twice as likely to benefit from treatment than those on Lumizyme (alglucosidase alfa), a new analysis of data from the Phase 3 COMET trial suggests. The analysis employed a method called the…
Higher doses of Lumizyme (alglucosidase alfa) than what’s approved may improve survival outcomes for children with classic infantile onset Pompe disease (IOPD), according to a new study. An analysis of data from the Pompe Registry collected over nearly two decades showed that IOPD patients given Lumizyme at higher…
A 1-year-old toddler with infantile-onset Pompe disease has normal heart and motor function and is growing well after receiving in-utero enzyme replacement therapy (ERT) with Lumizyme (alglucosidase alfa) and standard postnatal immunotherapy, according to a report. The successful outcome with Ayla is the result of the first in-utero use…
We are no strangers to our insurance company. While it’s been great in most aspects, we still have to battle it from time to time. Unfortunately, this happens quite often with rare disease patients. Due to his infantile-onset Pompe disease, my 3-year-old son, Cayden, has to…
Once a week, my 3-year-old son, Cayden, receives a six-and-a-half-hour infusion for his infantile-onset Pompe disease. I’ve previously shared a look inside his home infusions. However, some people wonder how we can keep my son entertained and content for such a long period of time. As Cayden has…
Although enzyme replacement infusions were initially very frightening, they have become a part of our routine. My son, Cayden, requires this lifesaving medication due to his infantile Pompe disease. It works by replacing the enzyme that his body cannot make on its own. For the first…
When administered early and above its recommended dose, Myozyme (alglucosidase alfa, known as Lumizyme in the U.S.) may improve the clinical outcomes and prognosis of people with infantile-onset Pompe disease (IOPD), a study reports. Safety with a higher dose did not seem to be a concern, the…
Pharmaceutical executives rarely make for a sympathetic Hollywood medical drama. But John Crowley did, and in the nearly 10 years since the release of “Extraordinary Measures” — a tearjerker starring Brendan Fraser as Crowley and Harrison Ford as short-tempered scientist Robert Stonehill — biotech has seen a huge transformation, both…
Pompe disease, an inherited lysosomal storage disorder, is much more common than once thought, say several experts speaking at the Muscular Dystrophy Association’s recent 2019 MDA Clinical and Scientific Conference in Orlando, Florida. For years, the incidence of Pompe was believed to be anywhere from 1 in…