Research Updates Spring 2012

Note: The research news featured in Research Updates is a compilation of Quest News Online articles posted between issues of the print magazine.

• Repligen Launches Italian Trial of Experimental Drug for FA, March 15
• Conference Provides Updates for MDA Clinic Team Members, March 9
• DMD: Eteplirsen Allows Production of Functional Dystrophin, February 8
• MDA Commits $12 Million to Neuromuscular Disease Research, February 1
• Jacobus Begins Invitation-Only Trial of 3,4-DAP in LEMS, January 25
• DUX4 Causes Muscle Mayhem in FSHD, January 19
• Immunosupression Induces Tolerance to Enzyme Treatment in Pompe Disease, January 16
• MDA Funds Development of Utrophin 'Magnet' for DMD/BMD, January 13
• Nationwide Children's Podcast Probes Exon Skipping in DMD, January 11
• Phase 1 Trial Opens of ISIS-SMNRx in Children with SMA, January 2
• Frataxin 'Fusion' Protein Rescues FA Mice, December 26
• MDA Funds Clinical Studies of Reformulated Drug for DMD, December 20
• Trial Opens of 'Pharmacological Chaperone' in Pompe Disease, December 19
• Podcast Explores Blocking NF-kappa B in DMD, December 14
• International MMD Consortium Includes Professionals, Families, December 8
• PTC, Roche Team Up Against SMA, December 1
• Seeking Participants for hIBM/GNE Clinical Trial, November 30

Also featured:

• Trials of DMD Exon-Skipping drug GSK2402968 Continue (link redirects to ClinicalTrials.gov)
  • A phase 2 trial in the U.S. (trial number: NCT01462292)
  • A phase 3 trial of walking DMD-affected boys in Canada, Europe and Asia (NCT01254019)
  • A phase 3 trial of boys in Europe and Japan who have been in previous GSK2402968 trials (NCT01480245)