Local child continues his battle with Pompe Disease

River Pugh, a three-year-old boy from Indiana, is fighting to survive Pompe Disease, a rare genetic disease that causes ...
WebMD

Managing Late-Onset Pompe Disease With Opfolda

Opfolda (miglustat) is a medicine taken with another medicine for certain people with late-onset Pompe disease. Pompe disease is a rare genetic disorder that is caused by an abnormal gene for a ...
Insider Monkey

Amicus Therapeutics Publishes Positive PROPEL Study Data for Pompe Disease Treatment

Amicus Therapeutics Inc. (NASDAQ:FOLD) is one of the best biotech stocks to invest in now. On June 3, Amicus Therapeutics announced the publication of a post-hoc analysis of data from the ...
Nasdaq

Amicus Therapeutics Publishes Positive Post-Hoc Analysis of Cipaglucosidase Alfa-Atga + Miglustat in Late-Onset Pompe Disease

Amicus Therapeutics has released a post-hoc analysis from the PROPEL study, published in Muscle and Nerve, which evaluated the efficacy of cipaglucosidase alfa-atga combined with miglustat (cipa+mig) ...
MarketWatch

Pompe Disease Market Research 2025: Treatment, Route of Administration, and Region Analysis and Forecast to 2035 Featuring Amicus Therapeutics, Astellas, BioMarin, Maze ...

The "Pompe Disease Market - A Global and Regional Analysis: Focus on Treatment Type, Route of Administration, and Region - Analysis and Forecast, 2025-2035" report has been added to ResearchAndMarkets ...
Nasdaq

Amicus Therapeutics' Pombiliti+Opfolda Receives Japan MHLW's Approval For Pompe Treatment

(RTTNews) - Amicus Therapeutics, Inc. (FOLD), Wednesday announced that Japan's Ministry of Health, Labour and Welfare or MHLW has approved the combination therapy of Pombiliti and Opfolda for the ...
Morningstar

Aro Biotherapeutics Completes Enrollment in Phase 1b Trial of Targeted siRNA Therapy, ABX1100, in Late-Onset Pompe Disease (LOPD)

Milestone marks progress toward first-ever demonstration of a targeted GYS1 substrate reduction therapy in Pompe disease Aro Biotherapeutics, a clinical-stage biotechnology company developing potent ...