Several cellular processes, particularly those related to lysosome function and autophagy, are altered in the muscles of people with late-onset Pompe disease (LOPD), and supplementation with an amino acid as a regulator could be a “promising therapeutic approach,” according to a study. Levels of L-arginine, an amino acid that…
Supplements
A diet combining an oral ketone precursor — a molecule being studied as a dietary supplement — and a cocktail of natural antioxidant molecules boosts the efficacy of enzyme replacement therapy (ERT) at maintaining muscle strength and motor function in a mouse model of Pompe disease, a study…
Adding L-alanine supplements to standard enzyme replacement therapy (ERT) for a young girl with Pompe disease led to improvements in several metabolic and body composition measures, a case report shows. According to researchers, this was the first known report of oral supplements of L-alanine — an amino acid,…
L-carnitine — a molecule often taken as an oral dietary supplement — boosts the efficacy of enzyme replacement therapy (ERT), increasing by four times the activity of acid alpha-glucosidase (GAA), the faulty enzyme in Pompe disease, in patient-derived cells, a study shows. The molecule was found to stabilize…
Antioxidants can increase the effectiveness of enzyme replacement therapy (ERT) for Pompe disease caused by oxidative stress, the damage in cells or tissues by reactive oxygen molecules, according to a study in cells and mice. The findings also showed that enhancing autophagy — the destruction of damaged or redundant…
Recent Posts
- 1-time gene therapy cuts need for enzyme treatments for Pompe children
- What a child with Pompe disease taught me about being a parent
- Switching therapies may stabilize walking decline in late-onset Pompe
- How I’m coping with mom guilt since my daughter’s Pompe diagnosis
- ERT helps keep breathing stable during sleep in pediatric Pompe disease