Avidity Biosciences announced this week that it had received FDA Orphan Drug Designation for its experimental FSHD treatment, called AOC 1020. This does NOT mean the drug is now available… Read More »
Avidity gets orphan drug designation
FDA Fast Track Designation for Avidity’s FSHD therapeutic
What does this mean for patients? An FDA Fast Track designation DOES NOT mean that the drug has been approved for use by patients. AOC 1020 is still a long… Read More »
(Cautious) optimism in FSHD drug development
Patients are critical in helping to transform optimism into reality by Ken Kahtava, Chief Business Officer, FSHD Society Breakthroughs in FSHD research have identified the primary mechanism that causes FSHD…. Read More »
Promising findings from Avidity’s myotonic dystrophy trial
A major breakthrough for the field of RNA therapeutics Avidity Biosciences announced today that its investigational treatment for myotonic dystrophy type 1 (DM1), called AOC 1001, can be delivered into… Read More »
Springboards to the future
Everything we do propels us faster toward treatments and a cure We often speak of “filling potholes” and “overcoming obstacles” toward treatments and cures, but what the FSHD Society has… Read More »