[…] (RNAi) therapeutic being developed as a potential treatment for patients with facioscapulohumeral muscular dystrophy ( FSHD). ARO-DUX4 is the first clinical candidate utilizing Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform […]
Arrowhead files for regulatory clearance to start a Phase 1/2 study in FSHD
Meet del-brax, Avidity’s FSHD therapeutic candidate
Results presented by Avidity of interim data from its Phase 1/2 clinical trial in FSHD were all the buzz at this year’s International Research Congress and FSHD Connect conference. […]
New biotech targets FSHD
Three entrepreneurs who have been affected personally by FSHD announced at this year’s FSHD Champions meeting that they have established a biotech dedicated to developing a treatment for FSHD. The new […]
Fruit fly model of FSHD is now available
[…] DUX4-fl gene, which encodes a paired homeobox domain transcription factor, is the primary mediator of FSHD pathology, but the mechanism is unknown. This is mainly due to the lack of […]
Acceleron to host webcast on FSHD clinical trial
[…] diseases, today announced that it will host an educational webinar on facioscapulohumeral muscular dystrophy ( FSHD) and the Company’s Phase 2 clinical trial of ACE-083 in FSHD patients. The webinar […]