Researchers from Arthex Biotech SL, the University of Valencia (Spain) and collaborators sought to address the lack of optimized microRNA (miRNA)-targeting antisense oligonucleotides (anti-miRs) for upregulating MBNL1 in myotonic dystrophy.
Arthex Biotech SL has received FDA clearance to initiate the phase I/IIa Arthemir study of ATX-01 for the treatment of myotonic dystrophy type 1 (DM1).
Arthex Biotech SA has achieved key regulatory milestones in its program to develop ATX-01 in myotonic dystrophy type 1 (DM1). Having held a pre-IND meeting with the FDA last year and received scientific advice from the EMA, Arthex plans to file an IND application in the U.S. and a clinical trial application (CTA) in Europe next year.
