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:
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:
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:
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:
Resources referenced in this webinar:
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:
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 MARINATM trial.
Presenters include:
Click here to find all our upcoming Meet the DM Drug Developers dates.
Presented on June 17th.
Do you have questions for DM experts? Join Sarah Howe, MBA, of the Marigold Foundation for an “Ask-the-Expert” discussion, A Community Portrait through Surveys and Insurance Claims Data!
The patient voice is critical to understanding myotonic dystrophy (DM). As experts in their own right, those living with DM can inform research, drug development, and government decision-making. Join Sarah Howe, MBA, Program Manager of the Marigold Foundation, as she presents findings from the Christopher Project and a DM insurance claims data analysis project. These studies shed new light on the burden of disease and illustrate the importance of patient-centered research in the development of scientific, clinical, and policy solutions to help improve the lives of individuals and families living with DM.
Speakers to include:
Sarah Howe, MBA has worked in myotonic dystrophy for 15 years as the Program Manager for the Marigold Foundation. Marigold is a private Canadian foundation focused on therapeutic development, research, and strategic initiatives in myotonic dystrophy. Most recently, Sarah was Project Coordinator for the Christopher Project, a large survey of patients, families, and caregivers living with DM1 and DM2 aimed at understanding their unmet needs and elevating the patient voice in myotonic dystrophy. Click here to read the open access publication.
Click here to find all our upcoming “Ask-the-Expert” question sessions!