Attendees at our FSHD Family Day conference in Baltimore this past Sunday enjoyed an excellent series of talks by the clinicians and researchers at the Kennedy Krieger Institute. Here are […]
Mid-Atlantic FSHD Family Day presentations
Human Muscle Growing in Mice Provides a New Research Tool for FSHD
[…] from the FSH Society, a patient-driven nonprofit, has enabled people with facioscapulohumeral muscular dystrophy ( FSHD) to donate muscle tissue, which scientists have succeeded in grafting into mice, providing a […]
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. […]
Arrowhead files for regulatory clearance to start a Phase 1/2 study in FSHD
[…] (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 […]
Ultragenyx Pharmaceutical to sponsor FSHD drug development project
[…] on the development of small molecule therapeutics for the potential treatment of Facioscapulohumeral Muscular Dystrophy ( FSHD). The SLU team is led by Fran Sverdrup, PhD, whose lab has been supported […]