Professor Alan H. Beggs
Harvard Medical School
Children´s Hospital Boston
300 Longwood Avenue, Boston, MA 02115, USA
Team#9, directed by Alan Beggs at the Children´s Hospital of Harvard University. An important asset of SARCOSI are the patients registries that have been established by Dr. Beggs. These registries include in particular patients with mutations in nebulin, and centronuclear myopathies where severe atrophy is a hallmark. The combination of clinical data, including response to therapies, and next generation sequencing is likely to pave the way not only to a better understanding of disease origins, but also their treatment.