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Rare Disease Day

Scientific Publications & Presentations

June 12-13, 2025FSHD International Research Conference

DYNE-302 Leads to Functional Improvement and Resolves Muscle Transcriptomic Changes in Mouse Models of FSHD

May 2-4, 2025Myotonic Dystrophy Foundation (MDF) Conference

Health Insurance Literacy in Myotonic Dystrophy Type 1 (DM1), An Interim Report
Changes in RNA Splicing as a Surrogate Endpoint for Myotonic Dystrophy Type 1 (DM1) Clinical Trials

March 16-19, 2025Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

Safety and Efficacy from the Ongoing Phase 1/2 DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping
Oral Presentation Poster
Safety and Efficacy of DYNE-101 in Adults with DM1: Phase 1/2 ACHIEVE Trial Data
Oral Presentation Poster
Characteristics of Patients with Myotonic Dystrophy Type 1 with Complex Care Needs: Results from the Real-World IMPaCT Study
The FORCE™ Platform Achieves Robust and Durable DUX4 Suppression and Improves Muscle Function in Facioscapulohumeral Muscular Dystrophy Mouse Model
The FORCE™ Platform Enables TfR1-mediated Delivery of Exon Skipping PMO to the CNS and Resolves Anxiety in a Mouse Model of DMD
Dyne-Sponsored Symposium: Harnessing the FORCE Platform to Advance Targeted Therapies for Neuromuscular Diseases

February 3-7, 202521st Annual WORLDSymposium™

The FORCE™ Platform Delivers Acid Alpha-Glucosidase to Muscle as well as Central Nervous System and Resolves Pathology in Pompe Disease Mice

January 18, 2025 Communications Medicine

FORCE™ Platform Overcomes Barriers of Oligonucleotide Delivery to Muscle and Corrects Myotonic Dystrophy Features in Preclinical Models

October 8-12, 2024 29th Annual Congress of the World Muscle Society

The FORCE™ Platform Enables TfR1-mediated Delivery of Enzyme Replacement Therapy to Muscle and Central Nervous System, Resolving Pompe Pathology in Mice
Initial Data from the ACHIEVE Trial of DYNE-101 in Adults with Myotonic Dystrophy Type 1 (DM1)
Initial Data from the DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping
The FORCE™ Platform Achieves Robust and Durable DUX4 Suppression and Improves Muscle Function in Facioscapulohumeral Muscular Dystrophy Mouse Model

June 23-26, 2024 New Directions in Biology and Disease of Skeletal Muscle Conference

FORCE™ Platform for the Development of Targeted Therapeutics for Rare Muscle Diseases

June 13-14, 202431st Annual FSHD Society International Research Congress

The FORCE™ Platform Achieves Robust and Durable DUX4 Suppression and Functional Benefit in FSHD Mouse Models

May 5-8, 2024ISPOR 2024

Costs and Healthcare Resource Utilization Evaluation in Myotonic Dystrophy Type 1: Results from the Real-world CARE-DM1 Study

April 13-18, 2024American Academy of Neurology (AAN) Annual Meeting

Initial Data from the DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping
Oral Presentation Poster

April 9-13, 202414th International Myotonic Dystrophy Consortium (IDMC) Meeting

Initial Data from the ACHIEVE Trial of DYNE-101 in Adults with Myotonic Dystrophy Type 1 (DM1)
Costs and Healthcare Resource Utilization Evaluation in Myotonic Dystrophy Type 1: Results from the Real-World CARE-DM1 Study

March 3-6, 2024Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

Initial Data from the DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping
Initial Data from the ACHIEVE Trial of DYNE-101 in Adults with Myotonic Dystrophy Type 1 (DM1)

February 16, 2024Parent Project Italy’s XXI International Conference on Duchenne and Becker Muscular Dystrophy

Patient engagement in clinical trial design for rare neuromuscular disorders: impact on the DELIVER and ACHIEVE clinical trials

January 2, 2024Research Involvement and Engagement

Patient engagement in clinical trial design for rare neuromuscular disorders: impact on the DELIVER and ACHIEVE clinical trials

May 17, 2023American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

The FORCE™ Platform Delivers Oligonucleotides to the Brain in a DM1 Mouse Model and in NHPs

May 7-10, 2023ISPOR 2023

The Humanistic Burden of Myotonic Dystrophy Type 1: A Literature Review

April 22-27, 2023American Academy of Neurology (AAN) Annual Meeting

ACHIEVE Trial, a Randomized, Placebo-Controlled, Multiple Ascending Dose Study of DYNE-101 in Individuals With Myotonic Dystrophy Type 1 (DM1)
Oral Presentation Poster

March 19-22, 2023Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

ACHIEVE Trial, a Randomized, Placebo-Controlled, Multiple Ascending Dose Study of DYNE-101 in Individuals With Myotonic Dystrophy Type 1 (DM1)
DELIVER, a Randomized, Double-blind, Placebo-Controlled, Multiple Ascending Dose Study of DYNE-251 in Boys With DMD Amenable to Exon 51 Skipping
FORCE™ Platform Achieves Robust Exon Skipping, Restores Dystrophin at the Sarcolemma and Halts Progression of Fibrosis in the Severe D2-mdx Model of DMD

October 11–15, 202227th International Hybrid Annual Congress of the World Muscle Society

Building a FORCE™ platform-based DMD franchise for the treatment of individuals with mutations amenable to exon skipping

August 10, 2022Nucleic Acids Research (NAR)

Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice

May 16, 2022American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

Repeat Dosing with DYNE-101 is Well Tolerated and Leads to a Sustained Reduction of DMPK RNA Expression in Key Muscles for DM1 Pathology in hTfR1/DMSXL Mice and NHPs

October 1, 20212021 Muscle Study Group Annual Scientific Meeting

FORCE™ Platform Delivers Exon Skipping PMO, Leads to Durable Increases in Dystrophin Protein in mdx Mice and Is Well Tolerated in NHPs

September 20-24, 2021World Muscle Society 2021 Virtual Congress

The FORCE™ Platform Achieves Durable Knockdown of Toxic Human Nuclear DMPK RNA and Correction of Splicing in the hTfR1/DMSXL Mouse Model.

June 25, 202128th Annual FSHD Society International Research Congress

FORCE™ Platform Enables Muscle-Targeted Delivery of Antisense Oligonucleotide and Silencing of DUX4 Activity in an FSHD Cell Line

May 14, 2021American Society of Gene & Cell Therapy Annual Meeting (ASGCT)

Splice Correction and Reduction of Toxic DMPK RNA In Vitro and In Vivo Utilizing Novel Antibody Targeted Antisense Oligonucleotides
The FORCE™ Platform Achieves Robust Knock Down of Toxic Human Nuclear DMPK RNA and Foci Reduction in DM1 Cells and in Newly Developed hTfR1/DMSXL Mouse Model

May 12, 2020ASGCT 2020

Targeted Delivery of ASOs Demonstrates Potential to Treat Duchenne Muscular Dystrophy
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