For most adults with late-onset Pompe disease (LOPD), a fairly significant improvement in walking ability is needed to be…
Articles by Marisa Wexler, MS
Most people with classic infantile-onset Pompe disease (IOPD) develop brain abnormalities despite treatment with Myozyme (alglucosidase alfa), and…
Throughout 2023, Pompe Disease News brought you coverage of the latest developments in research into Pompe disease…
Higher doses of Lumizyme (alglucosidase alfa) than what’s approved may improve survival outcomes for children with classic infantile onset…
The first healthy volunteer has been enrolled in a Phase 1 study testing ABX1100 — a novel substrate reduction therapy…
Adding the enzyme stabilizer miglustat did not improve the effectiveness of Lumizyme (alglucosidase alfa) or Nexviazyme (avalglucosidase alfa)…
Pombiliti (cipaglucosidase alfa) plus Opfolda (miglustat), a two-part therapy formerly known as AT-GAA, has been approved by the U.K.’s Medicines…
Some children with Pompe disease have dysfunctional motor neurons — the specialized nerve cells that control movement — in…
A newborn screening program for Pompe disease in Minnesota was found effective for getting babies with infantile-onset disease started…
After more than a decade on Lumizyme (alglucosidase alfa), a teenager with late-onset Pompe disease (LOPD) developed an…