LOPD

The six-minute walk distance (6MWD) test is a reliable measure of declines in motor abilities in people with late-onset Pompe disease (LOPD), a two-year study that evaluated a series of such tests reported. Other common measures of motor function and muscle strength failed to reliably detect such changes in…

Macroglossia, or an abnormally enlarged tongue, caused difficulty swallowing, speech abnormalities, and reduced breathing during sleep for five people with late-onset Pompe disease (LOPD), according to a recent French study. Treatment with enzyme-replacement therapy (ERT), a standard of care for Pompe patients, did not alleviate the debilitating symptoms,…

A program of supervised resistance exercise can improve muscle function — including in muscles needed to breathe — for adults with late onset Pompe disease (LOPD), a small study suggests. “Our findings encourage use of supervised exercise and inspiratory muscle training for improving physical function and health to improve…

The investigational gene therapy ACT-101 overall was well-tolerated among three people with late-onset Pompe disease (LOPD) who were treated in a Phase 1/2 clinical trial. Moreover, the trial results also showed that treatment markedly increased levels of the GAA enzyme — deficient in Pompe disease — in the patients’ muscle…

Interruptions to Myozyme enzyme replacement therapy (ERT) regimens during the COVID-19 pandemic led to worsened motor symptoms and poorer respiratory function in patients with Pompe disease, a French study found. These decreases in motor and respiratory function — seen after just a couple of months — were not…

Infection by the virus that causes COVID-19 led to mild-to-moderate symptoms in four people with late-onset Pompe disease (LOPD) and pulmonary involvement, despite the increased risk of severe complications in such cases, scientists reported. “COVID-19 infection can result in a benign course in some patients with LOPD. However, individuals with LOPD…

Long-term treatment with Nexviazyme (avalglucosidase alfa) safely and effectively improves lung function and walking ability in children and adults with late-onset Pompe disease (LOPD), according to nearly two years of data from the Phase 3 COMET trial. These findings, along with positive long-term results from the Phase 2…

Acceptable safety is being seen to date in four adults with late-onset Pompe disease (LOPD) given the one-time gene therapy  AT845 in the FORTIS Phase 1/2 trial, its developer, Astellas Gene Therapies, announced. Interim study data in these four enrolled patients, as of the Dec. 3 cutoff date, shows…

Long-term treatment with Myozyme (alglucosidase alfa) — marketed in the U.S. as Lumizyme — results in variable but satisfactory motor and respiratory outcomes among older adults recently diagnosed with late-onset Pompe disease (LOPD), a study from Germany reported. According to its researchers, these findings were…

Nexviazyme (avalglucosidase alfa), a next-generation enzyme replacement therapy, has been approved by Health Canada for people with late-onset Pompe disease ages 6 months and older. “The Health Canada approval of Nexviazyme is an important milestone for Canadian Pompe patients and may represent a new standard of care,” Mark…