Genea Biocells, a San Diego-based biotech company focused on drug development to treat neuromuscular diseases, today announced that it has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its therapeutic candidate, GBC0905, for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Scientists from Genea Biocells will be attending the FSH… Read More »
FDA orphan drug designation for Genea Biocells’ FSHD drug candidate
Walk Pioneers Lead the Way!
This September and early October, the FSH Society will host its new signature fundraising event, the Walk & Roll to Cure FSHD in five communities from coast to coast. These Volunteer Leaders are paving the way for their communities to raise funds and increase awareness! 5 Brave Walk Pioneers have agreed to attend monthly training sessions, beta-test… Read More »
DUX may be a master switch of the genome
The DUX4 gene in FSH muscular dystrophy is typically described as a rogue actor, a genetic oddball that is never supposed to be active in adult muscle, and is rendered harmless by an elaborate lockdown system. It is only when several parts of the “lock” mechanism break that DUX4 emerges to cause damage to muscles…. Read More »
Rochester FSHD Family Day videos
Watch the videos here. The University of Rochester Fields Center for FSHD Research has posted a summary and videos from the 6th annual FSHD Family Day Conference held on April 7, 2018. Jointly sponsored by the University of Rochester, Leiden University Medical Center, and the FSH Society, the meeting presented the latest developments in FSHD… Read More »
My journey from powerless to powerful
by Dave Lukas, Lake In the Hills, Illinois Three years ago, my life was changed forever. Three years ago, I walked out of a doctor’s office, got in my car and sobbed. Three years ago, my image of what my future looked like was shattered. Today, my life is still changed forever. Today, I leave… Read More »