This session provides an overview of the clinical trial process, helping attendees understand what to expect and how to prepare. It covers the different phases of trials, participant experiences, and key do’s and don’ts. Attendees will leave with the knowledge needed to make informed decisions about myotonic dystrophy trial participation.

Myotonic Dystrophy Industry Updates was presented in partnership by Euro-DyMA (European Dystrophia Myotonica Association) and the Myotonic Dystrophy Foundation (MDF) as a part of the 2025 MDF Conference on May 2nd and 3rd, 2025.

Click here to learn more about 2025 MDF Conference in Indianapolis, Indiana! >>>

In this session, representatives from biotech and pharma companies provide updates on their drug development efforts in the DM field.

Presenting organizations include:

• Avidity Biosciences
• Dyne Therapeutics
• ARTHEx Biotech
• Juvena Therapeutics
• Lupin Neurosciences
• PepGen
• Vertex Pharmaceuticals
• Sanofi
• Modalis Therapeutics
• Arrakis Therapeutics

Thank you to our 2025 MDF Conference Sponsors for helping make this event possible:

• Lead Sponsor: Avidity Biosciences
• Diamond Sponsors: Dyne Therapeutics and Arthex Biotech
• Platinum Sponsors: PepGen and Vertex Pharmaceuticals
• Gold Sponsor: Sanofi
• Bronze Sponsor: Lupin Neurosciences

Explore our other sessions and read about the incredible 2025 MDF Conference in Indianapolis, Indiana!

The 5th Edition of Pharma Day was presented in partnership by Euro-DyMA (European Dystrophia Myotonica Association) and the Myotonic Dystrophy Foundation (MDF) as a part of the Professional Track at the 2025 MDF Conference on May 1st, 2025.

Click here to learn more about 2025 MDF Conference in Indianapolis, Indiana! >>>

This program brings pharmaceutical innovators, researchers, clinicians, and patient organizations together for an afternoon of presentations and face-to-face discussions on drug development and research advancements.

Presenting organizations include:

• Euro-DyMA (European Dystrophia Myotonica Association)
• The Myotonic Dystrophy Foundation (MDF)
• The DMCRN (Myotonic Dystrophy Clinical Research Network)
• Modalis Therapeutics
• Sanofi
• Vertex Pharmaceuticals
• Arrakis Therapeutics
• Avidity Biosciences
• Dyne Therapeutics
• PepGen
• Lupin Neurosciences
• ARTHEx Biotech

Thank you to our Lead Sponsor, Avidity Biosciences, our Diamond Sponsors, Dyne Therapeutics, and Arthex Biotech, our Platinum Sponsors, PepGen, and Vertex Pharmaceuticals, Gold Sponsor, Sanofi, Bronze Sponsor, Lupin Neurosciences, and Arrakis Therapeutics for supporting Pharma Day at the 2025 MDF Conference.

Explore our other sessions and read about the incredible 2025 MDF Conference in Indianapolis, Indiana!

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!