Four children not making any acid maltase enzyme tolerated enzyme replacement therapy when also treated with immunosuppressants
posted on January 16, 2012 - 6:00am
Editor's note 2/2/12: This story was updated to reflect the award of a new MDA grant to Eric Sjoberg at Amicus Therapeutics.
Drugs that suppress the immune system can successfully prevent or reverse rejection of enzyme replacement therapy in Pompe disease (acid maltase deficiency), a new study has shown.
Isis Pharmaceuticals will test its experimental antisense drug in 24 children with spinal muscular atrophy at five U.S. centers
posted on January 2, 2012 - 6:00am
A 24-person, phase 1 trial to test the safety and tolerability of the experimental drug ISIS-SMNRx in children with spinal muscular atrophy (SMA) is now open at Columbia University Medical Center in New York, with additional sites expected to open in Boston, Philadelphia, Dallas and Salt Lake City.
MDA has awarded $750,000 to Summit Corp. to test a new formulation of its utrophin booster SMT C1100 for Duchenne muscular dystrophy
posted on December 20, 2011 - 12:05pm
MDA has awarded $750,000 to Summit Corporation PLC for development and testing of SMT C1100, the company's experimental drug for treatment of Duchenne muscular dystrophy (DMD). Summit is an Oxford, United Kingdom, drug discovery company.
The award was made through MDA's Venture Philanthropy (MVP) arm, a part of MDA's translational research program.
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
The biopharmaceutical company Amicus Therapeutics is testing its experimental drug AT2220 in combination with enzyme replacement therapy in people with Pompe disease (acid maltase disease, or AMD).
A phase 1 trial of Ultragenyx’ treatment for hereditary inclusion-body myopathy caused by GNE mutations is taking place in New York and California
posted on November 30, 2011 - 2:46pm
Ultragenyx Pharmaceutical in Novato, Calif., is developing an experimental treatment for a form of hereditary inclusion-body myopathy, or hIBM, that’s caused by mutations in the GNE gene.
This gene codes for a protein that’s needed to make sialic acid. The Ultragenyx product is an extended-release form of sialic acid.
AVI BioPharma is developing two new exon-skipping drugs; GlaxoSmithKline will conduct more trials of GSK2402968, for which participants are being recruited
posted on November 21, 2011 - 6:00am
Two pharmaceutical companies recently announced they are expanding their development and testing of exon-skipping drugs for Duchenne muscular dystrophy (DMD).
A drug approved in Europe to treat Lambert-Eaton myasthenic syndrome is now being tested in the United States; clinical trial participants sought
posted on November 18, 2011 - 6:00am
BioMarin Pharmaceutical of Novato, Calif., is conducting a multicenter study of 3,4-diaminopyridine phosphate (3,4-DAP), also known as amifampridine phosphate, in adults with Lambert-Eaton myasthenic syndrome (LEMS).
There are eight U.S. trial sites, with additional sites planned for France, Germany, Italy, Poland and Spain.
