Throughout 2023, Pompe Disease News brought you coverage of the latest developments in research into Pompe disease…
Articles by Marisa Wexler, MS
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…
The enzyme stabilizer Opfolda (miglustat), one part of the two-component therapy formerly known as AT-GAA (cipaglucosidase alfa/miglustat), has been…
The switch to at-home infusions of enzyme replacement therapy (ERT) — made necessary due to the COVID-19 pandemic —…