Shots:
The US FDA has granted FTDto ATX-01 for the treatment of Myotonic Dystrophy Type 1 (DM1)
ATX-01 showed increased free MBNL, improved splicing abnormalities, & restored function in animal models, & is being evaluated in the P-I/IIa (ArthemiR) study
ATX-01 is an RNA-based therapeutic designed to inhibit miR-23b, which results in increased MBNL protein…
Shots:
The US FDA has granted FTD to DYNE-101 for treating myotonic dystrophy type 1 (DM1). US Accelerated Approval is anticipated to submit in H1’26
DYNE-101 is being tested in the global P-I/II ACHIEVE trial for DM1
DYNE-101 is an antisense oligonucleotide (ASO) linked to an antibody fragment (Fab) that targets the transferrin…

