A powerful imaging technique called high-resolution intravital microscopy, or IVM, may be a useful tool for visualizing and quantifying the effectiveness of investigational treatments in mouse models of Pompe disease, according to recent research. The approach allows for cellular changes in muscles to be visualized in live animals. Using…
treatment
My 6-year-old son, Cayden, is no stranger to ear infections. When he was 2, we decided it was time to solve the problem of recurring ear infections once and for all. His otolaryngologist recommended implanting ear tubes. Tubes are pretty common, so I wasn’t overly concerned about the…
Sanofi’s Nexviazyme (avalglucosidase alfa), an enzyme replacement therapy approved for Pompe disease, doesn’t appear to be linked to new or worsening diabetes, a concern raised by the presence of dextrose, a simple sugar, in the solution for infusion. In a study funded by Sanofi, researchers also…
Using self-reported outcomes by adults with Pompe disease, researchers were able to estimate the minimal clinically important difference or MCID — the smallest outcome change that a patient perceives as important following treatment — after one year of enzyme replacement therapy (ERT). The differences reported by patients as clinically…
Long-term treatment with Pombiliti plus Opfolda (cipaglucosidase alfa/miglustat) safely improved motor function and stabilized or enhanced lung function in adults with late-onset Pompe disease (LOPD), according to newly published data from two clinical trials. Pompe disease is marked by a deficiency in an enzyme called acid alpha-glucosidase…
Infantile-onset Pompe disease (IOPD) patients who develop antibodies against enzyme replacement therapy (ERT) exhibit a distinct immune profile from those who don’t, according to recent research. Patients who developed these so-called high and sustained antibody titers, or HSAT, were skewed toward a type 2 immune profile, which the…
Philips, a company that manufactures and sells respiratory equipment to a big portion of the United States, recently posted a statement saying it was discontinuing “the sale of hospital ventilation products, certain home ventilation products, portable and stationary oxygen concentrators, and sleep diagnostic products.” This change was a shock…
Amicus Therapeutics has been honored with the WORLDSymposium 2024 New Treatment Award for Pombiliti + Opfolda (cipaglucosidase alfa/miglustat), the first medication duo to be approved for adults with late-onset Pompe disease. The award recognizes new treatments that received regulatory approval for lysosomal diseases like Pompe disease…
Switching from Lumizyme (alglucosidase alfa) to Nexviazyme (avalglucosidase alfa) was shown to improve breathing and motor function for most people with late-onset Pompe disease (LOPD) in a small real-world study. Many of the improvements, such as those seen in walking ability, were minimal, and did not reach…
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…
Recent Posts
- Home enzyme replacement therapy safe for Pompe disease children
- What ‘lucky’ really means for a child with Pompe disease
- Infantile Pompe brain changes may begin long before signs appear
- 1-time gene therapy cuts need for enzyme treatments for Pompe children
- What a child with Pompe disease taught me about being a parent