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

Presented on September 9th, 2023.

John Day, MD, PhD
Stanford

Charles Thornton, MD
University of Rochester Medical Center

Hear about the significant progress in the DM field from leading experts during this panel discussion. Learn about the impact the community can have on continuing the progress toward treatments and a cure.

Click here to learn more about the 2023 MDF Annual Conference.

Presented on September 9th, 2023.

With an opening statement from Dr. Andy Berglund, MDF Scientific Advisory Committee member, representatives from biotechnology and pharmaceutical companies will provide updates on their drug development efforts in the DM field.

Presenters include:

• Dyne Therapeutics
• PepGen
• Harmony Biosciences
• Rgenta Therapeutics
• AMO Pharma

Click here to learn more about the 2023 MDF Annual Conference.

Presented on September 8th, 2023.

With an opening statement from Dr. Andy Berglund, MDF Scientific Advisory Committee member, representatives from biotechnology and pharmaceutical companies will provide updates on their drug development efforts in the DM field.

Presenters include:

• Avidity Biosciences
• Juvena Therapeutics
• Sanofi
• ARTHEx Biotech

Click here to learn more about the 2023 MDF Annual Conference.

Presented on September 8th, 2023.

Gain insight and understanding on the important role the FDA plays in the drug approval process through a presentation from the FDA. This informative presentation will be followed by a panel discussion with the FDA, a DM expert clinician, and members of the DM Community. This session provides an opportunity for our community and clinicians to share the serious medical challenges facing our community with the FDA.

Speakers include:

• Nicholas Johnson, MD, MSci., FAAN, Virginia Commonwealth University
• Michelle Campbell, PhD, Federal Drug Administration
• Ami K. Mankodi, MD, Federal Drug Administration
• Kevin Brennan, MDF Advocacy Consultant
• Mark Planco
• Jeannine DeSoi
• Haley Martinelli

Click here to learn more about the 2023 MDF Annual Conference.

Presented on September 15, 2023.

A special webinar presentation in honor of September 15th, 2023, the 3rd annual International Myotonic Dystrophy Awareness Day! Learn how you can take action today to increase DM Awareness around the world and to change the future of myotonic dystrophy (DM).

Presentations include:

• The Importance of Patient Registries for Improving Clinical Trial Readiness
  Sofia Olmos, PhD, Myotonic Dystrophy Family Registry Coordinator
   
• MDF’s 2023 Advocacy Efforts on Capitol Hill
  Kevin Brennan, MDF Advocacy Consultant

Resources referenced in this webinar:

• The official International Myotonic Dystrophy Awareness Day website.
   
• Myotonic Dystrophy Family Registry Information.
   
• Myotonic Dystrophy Foundation Advocacy Day Talking Points, plus information on Federal Legislative Priorities.
   
• 2023 Template for contacting your Representatives.
   
• Downloadable Social Media Toolkits for spreading DM Awareness.
   
• Downloadable Myotonic Dystrophy Resources, including Anesthesia Guidelines, Exercise Guide, and more!
   
• Deutschsprachige Ressourcen zur Myotonen Dystrophie.
   
• Recursos en español para distrofia miotónica.

In addition to the International Myotonic Dystrophy Awareness Day “Call to Action” to urge Congress to support federal funding for myotonic dystrophy research, if you are interested in volunteering to become more involved in MDF advocacy, please email Kevin Brennan at kbrennan@bluebird-strategies.com. He is recruiting interested advocates who want to build awareness and action in Congress on behalf of our 2024 legislative priorities. As an advocate you’ll be invited to participate in several advocacy training webinars and efforts to recruit 15-20 family and friends to email, write, call, or visit our Senators and Representatives next year to support research funding.

Please share this video link with your friends and family to help celebrate International Myotonic Dystrophy Awareness Day!

We also encourage you to share your experiences with myotonic dystrophy on social media, and talk to members of your community — your friends, family, clinical care teams, classmates, and colleagues — about your experiences with DM.

Be sure to post with (and follow) the official International Myotonic Dystrophy Awareness Day hashtags #myotonicDystrophy and #MyotonicDystrophyAwareness to see what your fellow community members around the world are up to!

Thank you all for helping to change the future of myotonic dystrophy!

Presented on September 10, 2022.

Presented by Jacinda Sampson, MD of Stanford University and Kevin Brennan of Bluebird Strategies

Learn about biobanks, natural history studies, clinical trials, registries, and other types of research, the role that they play in drug development, and how DM1 and DM2 families can participate right now. Hear how MDF has and continues to facilitate research towards a cure, and discover ways that you can help increase federal funding for DM research right now!

Click here to learn more about the 2022 MDF Annual Conference.

Presented on August 5th, 2022.

Each month 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 learn more about PepGen.

Speakers include:

• Alayna Tress, MPH, Associate Director of Patient Advocacy
• Michelle Mellion, MD, Senior Vice President & Head of Clinical Development
• Jaya Goyal, PhD, Executive Vice President of Research & Preclinical Development
• Holly Hand, Clinical Operations Consultant
• Jane Larkindale, DPhil, Vice President of Clinical Science

Click here to find all our upcoming Meet the DM Drug Developers dates.

Presented on July 22nd, 2022.

Each month 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 learn more about Harmony Biosciences.

Learn more about their ongoing clinical trial, "Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1" on ClinicalTrials.gov.

Speakers include:

Rachel Radomski, Patient Advocacy Team
As the head of Patient Advocacy, Rachel Radomski leads Harmony’s work with people living with rare diseases, their caregivers and patient advocacy organizations across the rare disease landscape, including the Myotonic Dystrophy Foundation. The Patient Advocacy team works to represent the voice of people living with a rare disease across Harmony and develops innovative solutions and programs to meet the dynamic needs of the communities Harmony serves. Rachel and her team are honored to live Harmony’s core value of keeping “patients at the heart” of everything we do at Harmony Biosciences.

Dr. Bill Jacobson, Clinical Team
William Jacobson is the Senior Director, Clinical Development at Harmony Biosciences. Dr. Jacobson is an experienced Drug Developer and Clinical Trialist, originally trained as a Neuroscientist, with almost 30 years of experience in the pharmaceutical industry. Dr. Jacobson works on strategic and tactical aspects of drug development and his current focus is on rare diseases of the Central Nervous System. He is the clinical lead on Harmony Biosciences development program in type 1 myotonic dystrophy.

Click here to find all our upcoming Meet the DM Drug Developers dates.

Presented on July 1st, 2022.

Each month 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 learn more about Avidity Biosciences.

The Avidity Biosciences team presents on their AOC technology, the progress of their AOC 1001 program for DM, and the ongoing MARINA^TM trial.

Presenters include:

• Alissa Peters, Patient Advocacy
• Li Tai, MD. PhD., Clinical Development
• Husam Younis, Pharm.D., Ph.D., Research
• Ben Knisely, B.S., Clinical Operations
• Joe Johnston, Regulatory
• Carolyn Ball, P.E., Manufacturing

Click here to find all our upcoming Meet the DM Drug Developers dates.