enzyme replacement therapy

Drug testing for Duchenne MD, Leigh syndrome and Pompe disease moves forward; and two MDA-supported mouse studies suggest leads for myasthenia gravis and periodic paralysis

posted on March 26, 2013 - 4:21pm
Drug development and identifying new leads for possible drug development are in the news for five neuromuscular diseases in MDA’s program.

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.

A study of 11 long-term survivors of infantile-onset Pompe disease shows enzyme treatment can provide significant benefits, but deficits remain

posted on October 4, 2012 - 5:00am
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

posted on June 28, 2012 - 2:11pm
PTC begins non-US study of ataluren in DMD/BMD
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