Novartis Reports del-desiran P-III HARBOR Study Data in Myotonic Dystrophy Type 1

Shots: Novartis announced that the P-III HARBOR study of delpacibart etedesiran (del-desiran) in people with myotonic dystrophy type 1 (DM1) did not meet its 1EP of video hand opening time (vHOT) Del-desiran showed evidence of clinical activity in 2EPs and exploratory analyses, while safety findings were generally consistent with previously reported data; Novartis is evaluating…

ByPrince GiriSeptember 8, 2026

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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…

ByRidhi RastogiMarch 11, 2026

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Dyne Therapeutics’ DYNE-101 Secures the US FDA’s Fast Track Designation for Treating DM1

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…

ByDipanshu DixitJanuary 27, 2025

Novartis Reports del-desiran P-III HARBOR Study Data in Myotonic Dystrophy Type 1

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

Dyne Therapeutics’ DYNE-101 Secures the US FDA’s Fast Track Designation for Treating DM1

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