ARTHEx Biotech’s ATX-01 Secures the US FDA Fast Track Designation for Myotonic Dystrophy Type 1

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 expression, & reduced foci formation & toxic DMPK mRNA in DM1 pts

Ref: PRnewswire | Image: ARTHEx Biotech | Press Release

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