Amicus Therapeutics' experimental drug AT2220 has enhanced enzyme replacement therapy in a phase 2 trial in Pompe disease and is slated for further development

posted on February 15, 2013 - 4:29pm
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

posted on October 12, 2012 - 3:21pm
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.

Enrollment is open for a trial of AT2220, a drug designed to enhance the effects of enzyme replacement therapy in people with acid maltase deficiency

posted on December 19, 2011 - 1:10pm

News briefs about research in Charcot-Marie-Tooth disease, hereditary inclusion-body myositis, limb-girdle muscular dystrophy, myotubular/centronuclear myopathies and Pompe disease

posted on March 31, 2011 - 10:20am
Charcot-Marie-Tooth disease
posted on January 1, 2009 - 4:36pm
QUEST Vol. 16, No. 1
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
posted on July 1, 2008 - 1:37pm
QUEST Vol. 15, No. 4
This article includes items on: Duchenne muscular dystrophy, myotubular myopathy, acid maltase deficiency, spinal muscular atrophy, genetic information, limb-girdle muscular dystrophy, congenital muscular dystrophy, translational research