Overall, long-term alglucosidase alfa treatment markedly extended survival as well as ventilation-free survival and improved cardiomyopathy. Pompe disease is characterized by a deficiency of acid ...

Treatment also improved indices of cardiomyopathy, motor skills, and functional independence. Pompe disease is an autosomal-recessive disorder resulting from a lysosomal acid α-glucosidase (GAA ...

"Shionogi secures licence for Maze’s Pompe disease treatment" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been ...

GeneVentiv Therapeutics, a gene therapy company, today announced it has signed a global licensing agreement with Duke ...

The US regulator has approved Nexviazyme (avalglucosidase alfa) for the treatment of patients aged one year of age and older with late-onset Pompe disease, which progressively attacks the heart ...

which like Pompe, is a glycogen storage disease. Chief executive Bradley Campbell said that once both components of the treatment regimen are approved, "we believe there is significant commercial ...

Aro Biotherapeutics has dosed the first patient in a Phase Ib trial of ABX1100, a novel treatment for late-onset Pompe disease (LOPD), a rare inherited disorder that causes progressive muscle ...

Koeberl previously invented a gene therapy limited to treating late-onset Pompe disease that was licensed to AskBio. For nearly two decades, Dr. Koeberl has been working toward a treatment that ...

increased investments in rare disease treatments, better patient access to innovative therapies due to improvements in healthcare infrastructure, FDA approval of Nexviazyme for late-onset Pompe ...