Pompe disease results from a deficiency of an enzyme called acid-alpha-glucosidase (GAA), leading to massive lysosomal glycogen buildup in striated muscle, resulting in respiratory failure ...

The therapy has been designated GENV-002. Pompe disease results from a deficiency of an enzyme called acid-alpha-glucosidase (GAA), leading to massive lysosomal glycogen buildup in striated ...

(MENAFN- PR Newswire) Pompe disease results from a deficiency of an enzyme called acid-alpha-glucosidase (GAA), leading to massive lysosomal glycogen buildup in striated muscle, resulting in ...

Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its latest therapy for the rare, inherited disorder. The US regulator has approved ...

Your mood plummets as you remember ticks spread Lyme disease. Lyme disease gained notoriety when cases spiked in 2022 and 2023, over doubling between 2017 and 2023, according to the CDC.

Two significant programs that invested in research on diabetes, dementia, obesity and kidney disease have ended since the start of the Trump administration. By Gina Kolata Robert F. Kennedy Jr ...

It offers oral therapy for adults with Fabry disease, and a unique two-part treatment designed for adults with late-onset Pompe disease. FOLD ranks 3rd on our list of the best Guru stocks.

ALS drug effectively treats Alzheimer's disease in new animal study NU-9 acts on a common mechanism at play in both neurodegenerative diseases Date: April 8, 2025 Source: Northwestern University ...

But when your dad lost control of his body because of a rare neurodegenerative disease, and died a month after diagnosis, your body forgets. Needs reminding. Even almost three years later.