Presented on February 7, 2025.

Dyne Therapeutics presents updates on their DM1 program & new clinical data from their ongoing phase 1/2 ACHIEVE trial. Click here to learn more about Dyne Therapeutics.

Presenters include:

• Ashish Dugar, PhD, MBA, Chief Medical Affairs Officer
• Katherine Beaverson, MS, Vice President of Global Patient Advocacy

MDF Resources referenced in the Video:

• Toolkits & Publications
• MDF Support Groups
• Digital Academy
• Calendar of Events
• DM Drug Development Pipeline
• Myotonic Dystrophy Family Registry (MDFR)
• 2025 MDF Annual Conference
• Sign up for My Dispatch, our monthly e-newsletter

About Meet the Myotonic Dystrophy Drug Developers

Biotechnology, pharmaceutical and academic partners, large and small, working on treatments and a cure for myotonic dystrophy will sit down with our community to share their progress and answer your questions. Click here to find all our upcoming Meet the DM Drug Developers dates!

Presented on November 22, 2024.

Lupin Neurosciences presents Mexiletine program myotonic dystrophy type 1 (DM1)-01 program. Click here to learn more about Lupin Neurosciences.

Presenters include:

• Mariska van Aswegen, Vice President – Neurology, EMEA
• Claus J. Jepsen, President, Global Specialty
• Alla Zozulya Weidenfeller, PhD, Senior Medical Director, Head of Medical Affairs and Clinical Development

MDF Resources referenced in the Video:

• Toolkits & Publications
• MDF Support Groups
• Digital Academy
• Calendar of Events
• DM Drug Development Pipeline
• Myotonic Dystrophy Family Registry (MDFR)
• 2025 MDF Annual Conference
• Sign up for My Dispatch, our monthly e-newsletter

About Meet the Myotonic Dystrophy Drug Developers

Biotechnology, pharmaceutical and academic partners, large and small, working on treatments and a cure for myotonic dystrophy will sit down with our community to share their progress and answer your questions. Click here to find all our upcoming Meet the DM Drug Developers dates!

Presented on October 4, 2024.

Arthex Biotech presents myotonic dystrophy program, including ATX-01 program for DM1 and an overview of the ArthemiR trial. Click here to learn more about ARTHEx Biotech.

Find the latest updates to the Arthex's ArthemiR Trial, including trial site recruiting status, on ClinicalTrials.gov.

Presenters include:

• Beatriz Llamusi, PhD, Chief Scientific Officer & Co-Founder, Arthex Biotech
• Judy Walker, MD, FRCP(C), Chief Medical Officer, Arthex Biotech

MDF Resources referenced in the Video:

• Toolkits & Publications
• MDF Support Groups
• Digital Academy
• Calendar of Events
• DM Drug Development Pipeline
• Myotonic Dystrophy Family Registry (MDFR)
• 2025 MDF Annual Conference
• Sign up for My Dispatch, our monthly e-newsletter

About Meet the Myotonic Dystrophy Drug Developers

Biotechnology, pharmaceutical and academic partners, large and small, working on treatments and a cure for myotonic dystrophy will sit down with our community to share their progress and answer your questions. Click here to find all our upcoming Meet the DM Drug Developers dates!

Presented at the MDF 2024 Regional Conference in Boston, MA, on Saturday, April 6, 2024 at the Le Méridien Boston Cambridge hotel in Boston, MA.

As academic and industry partners work to develop treatments and a cure for myotonic dystrophy (DM), it is more important now than ever that the DM community is ready to participate in trials. This talk provides an overview of clinical trials and help attendees think about how to prepare for upcoming trials and studies.

Speaker: Seward Rutkove, MD Department of Neurology, Beth Israel Deaconess Medical Center (BIDMC)

Click here to learn more about the 2024 MDF Regional Conference in Boston, MA!

Presented at the 2024 MDF Regional Conference on Saturday, June 1, 2024 at the Westin Seattle in Seattle, WA.

Representatives from biotechnology and pharmaceutical companies will provide updates on their drug development efforts in the DM field.

Representatives: Avidity Biosciences, Dyne Therapeutics, ARTHEx Biotech, Juvena Therapeutics, AMO Pharma, PepGen

Click here to learn more about the 2024 MDF Regional Conference in in Seattle, WA!

Presented at the 2024 MDF Regional Conference on Saturday, May 4, 2024 at the Dunn Tower at Houston Methodist in Houston, TX.

As academic and industry partners work to develop treatments and a cure for myotonic dystrophy (DM), it is more important now than ever that the DM community is ready to participate in trials. This talk providex an overview of clinical trials and help attendees think about how to prepare for upcoming trials and studies.

Speakers: Ericka P. Greene, MD, FAAN, MACM Director, Neuromuscular & Myotonic Dystrophy Clinics, Houston Methodist

Click here to learn more about the 2024 MDF Regional Conference in in Houston, TX!

Presented on August 2, 2024.

PepGen presents myotonic dystrophy program, including PGN-EDODM1 program and nonclinical data and an overview of the FREEDOM-DM1 trial. Click here to learn more about PepGen.

Find the latest updates to the PepGen trial, including trial site recruiting status, on ClinicalTrials.gov.

Presenters include:

• Jane Larkindale, DPhil, Vice President of Clinical Science, PepGen
• Alayna Tress, MPH, Director of Patient Advocacy, PepGen
• Johanna Hamel, MD, Assistant Professor of Neurology, Pathology & Laboratory, University of Rochester Medical Center

MDF Resources referenced in the Video:

• Toolkits & Publications
• MDF Support Groups
• Digital Academy
• Calendar of Events
• DM Drug Development Pipeline
• Myotonic Dystrophy Family Registry (MDFR)
• 2025 MDF Annual Conference
• Sign up for My Dispatch, our monthly e-newsletter

About Meet the Myotonic Dystrophy Drug Developers

Biotechnology, pharmaceutical and academic partners, large and small, working on treatments and a cure for myotonic dystrophy will sit down with our community to share their progress and answer your questions. Click here to find all our upcoming Meet the DM Drug Developers dates!

Presented on July 19, 2024.

Juvena Therapeutics presents on their myotonic dystrophy program, focusing on JUV-161, a therapeutic protein that has demonstrated the ability to restore muscle differentiation, promote survival, increase strength, and improve metabolism by restoring protein signaling mechanisms that are crucial for muscular and metabolic homeostasis. Click here to learn more about Juvena Therapeutics.

Presenters include:

• Hanadie Yousef, PhD, Chief Executive Officer & Co-Founder, Juvena Therapeutics
• Jeremy O'Connell, PhD, Chief Scientific Officer & Co-Founder, Juvena Therapeutics
• Bernard (Barney) King, MD, MBA, Chief Medical Officer, Juvena Therapeutics
• John Day, MD, PhD, Scientific & Clinical Advisor, Juvena Therapeutics; and Professor of Neurology, Pediatrics (Genetics) and Pathology, Director of the Division of Neuromuscular Medicine at Stanford University

MDF Resources referenced in the Video:

• Toolkits & Publications
• MDF Support Groups
• Digital Academy
• Calendar of Events
• DM Drug Development Pipeline
• Myotonic Dystrophy Family Registry (MDFR)
• 2025 MDF Annual Conference
• Sign up for My Dispatch, our monthly e-newsletter

About Meet the Myotonic Dystrophy Drug Developers

Biotechnology, pharmaceutical and academic partners, large and small, working on treatments and a cure for myotonic dystrophy will sit down with our community to share their progress and answer your questions. Click here to find all our upcoming Meet the DM Drug Developers dates!

Presented on July 12, 2024.

Dr. Ashish Dugar, Chief Medical Affairs Officer at Dyne Therapeutics, presents on new DYNE-101 ACHIEVE Clinical Trial Data. Click here to learn more about Dyne Therapeutics.

Biotechnology, pharmaceutical and academic partners, large and small, working on treatments and a cure for myotonic dystrophy will sit down with our community to share their progress and answer your questions. Click here to find all our upcoming Meet the DM Drug Developers dates!

MDF Resources referenced in the Video:

• Toolkits & Publications
• MDF Support Groups
• Digital Academy
• Calendar of Events
• DM Drug Development Pipeline
• Myotonic Dystrophy Family Registry (MDFR)
• 2025 MDF Annual Conference
• Sign up for My Dispatch, our monthly e-newsletter

Presented on March 15, 2024.

Join MDF for a Meet the Myotonic Dystrophy Drug Developers webinar with Avidity Biosciences! Don't miss this special presentation on the "Global Phase 3 HARBOR Study & Long-term MARINA-OLE Data". Click here to learn more about Avidity Biosciences.

Panelists include:

• Sarah Boyce, President & CEO, & Member of the Board of Directors, Avidity Biosciences
• Li-Jung Tai, MD, PhD, Executive Director in Clinical Development, Avidity Biosciences
• Kath Gallagher, Senior Vice President and Global Program Head, Myotonic Dystrophy Type 1, Avidity Biosciences
• John Day MD, PhD, Professor of Neurology, Pediatrics (Genetics) and Pathology, and Director of the Division of Neuromuscular Medicine, Stanford School of Medicine

About Meet the DM Drug Developers

Biotechnology, pharmaceutical and academic partners, large and small, working on treatments and a cure for myotonic dystrophy will sit down with our community to share their progress and answer your questions. Click here to find all our upcoming Meet the DM Drug Developers dates!

MDF Resources referenced in the Video:

• Toolkits & Publications
• MDF Support Groups
• Digital Academy
• Calendar of Events
• 2024 MDF Regional Conferences
• Sign up for My Dispatch, our monthly e-newsletter