Amicus Therapeutics' experimental drug AT2220 has enhanced enzyme replacement therapy in a phase 2 trial in Pompe disease and is slated for further development
The experimental drug AT2220 has shown benefit as an enhancer of enzyme replacement therapy for the metabolic muscle disorder Pompe disease (acid maltase deficiency).
The drug, a pharmacological chaperone, is designed to:
Encouraging results have been reported for the first three dosage groups in a phase 2 trial of the 'pharmacological chaperone' AT2220
Biopharmaceutical company Amicus Therapeutics presented updated and encouraging results for its experimental Pompe disease (acid maltase deficiency) compound AT2220 this week at the 17th International Congress of the World Muscle Society in Perth, Australia.
A study of 11 long-term survivors of infantile-onset Pompe disease shows enzyme treatment can provide significant benefits, but deficits remain
A study of 11 children with infantile-onset Pompe disease (acid maltase deficiency) who started enzyme replacement therapy by the time they were 6 months old has shown the treatment can markedly improve the course of the disease, but that residual deficits persist.
New drug trials in Becker and Duchenne muscular dystrophies are open, and a new strategy to improve Pompe disease treatment is proposed
PTC begins non-US study of ataluren in DMD/BMD
This article contains items about: Pompe disease (acid maltase deficiency), Lambert-Eaton myasthenic syndrome, Duchenne and Becker muscular dystrophies, mitochondrial myopathy, myasthenia gravis and spinal muscular atrophy