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Digital Academy: Clinical Trials and Drug Approval

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Ionis Pharmaceuticals - Industry Updates On Drug Development (2018 MDF Annual Conference)

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

Speaker:
Laurence Mignon, PhD, IONIS Pharmaceuticals

PowerPoint Presentation

Click here for more presentations and videos from the 2018 MDF Annual Conference in Nashville, TN.

10/4/18 video
Accelerating The Search For Therapies: What's Happening, What's Next (2018 MDF Annual Conference)

Dr. Elizabeth Ackermann reports on the results of the 3-year MDF and Wyck Foundation-funded drug development acceleration effort, MDF 3.0, and introduces MDF’s new initiative, MDF 4.0, including the $1M gene editing project, the Myotonic Dystrophy Clinical Research Network study and other aggressive therapy development programs.

PowerPoint Presentation

Click here for more presentations and videos from the 2018 MDF Annual Conference in Nashville, TN.

10/4/18 video
Locana - Industry Updates On Drug Development (2018 MDF Annual Conference)

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

Speaker:
Ranjan Batra, PhD, Locana

PowerPoint Presentation

Click here for more presentations and videos from the 2018 MDF Annual Conference in Nashville, TN.

10/4/18 video
Expansion Therapeutics - Industry Updates On Drug Development (2018 MDF Annual Conference)

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

Speaker:
Matthew Disney, PhD, Expansion Therapeutics

PowerPoint Presentation

Click here for more presentations and videos from the 2018 MDF Annual Conference in Nashville, TN.

10/4/18 video
AMO Pharma Ltd. - Industry Updates On Drug Development (2018 MDF Annual Conference)

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

Speaker:
Joseph P. Horrigan, MD, AMO Pharma Ltd.

PowerPoint Presentation

Click here for more presentations and videos from the 2018 MDF Annual Conference in Nashville, TN.

10/4/18 webinar
Advocacy 101: What's Going on In Washington & How You Can Be an Effective Advocate

Adovocacy 101: What's Going on In Washington & How You Can Be an Effective Advocate by Jenn Dale of Faegre Baker Daniels Consulting.

For the first time ever, in 2017, myotonic dystrophy was recognized by leaders in Congress as an eligible research area in the Peer Reviewed Medical Research Program. We’d like to keep the momentum going by providing a legislative update to you, our network of advocates, and encouraging you to keep up that good work by continuing to engage with your Representatives and Senators. We’ll also be sharing some tips and tools that can be useful in your outreach. Topics that we will cover include: 
• What’s going on in Washington 
• MDF’s 2018 legislative agenda 
• Why the constituent voice matters most 
• How to be an effective advocate in your home state/district 
• How to build and strengthen relationships with your Representatives and Senators

7/11/18 video
MDF Research Update: What's Been Accomplished, What's Next

Dr. John Porter, PhD, Chief Science Officer at the Myotonic Dystrophy Foundation, reports out on the impact achieved and work carried out to date for the three year, $5M MDF 3.0 initaitive to accelerate drug development for DM, and what MDF is planning for our next drug development acceleration initiative, MDF 4.0. From the 2017 MDF Annual Conference.

4/16/18 video
Expanding the Pipeline: Industry Updates on Drug Development

Dr. Laury Mignon, from Ionis Pharmaceuticals, and Dr. Joseph Horrigan from AMO Pharma, report on the status of drug development efforts for DM patients. Jeremy Kelly and Dr. John Porter of the Myotonic Dystrophy Foundation introduce the session with a short overview on MDF's efforts to engage industry in DM therapy development. From the 2017 MDF Annual Conference.

4/16/18 video
Bringing the Patient Voice to CNS Targeting Drug Development in DM

MDF community members reported on the impact of myotonic dystrophy on the brain from their individual perspectives as people living with DM1, DM2 and as caregivers. Additional insights and comments were provided by members of the conference audience. The session input will be published and used to help drug developers understand the impact of DM on the brain from the patient perspective, and begin to identify clinical trial endpoints. From the 2017 MDF Annual Conference.

4/16/18 video
Bringing the Patient Voice to CNS Targeting Drug Development 2: Industry Observations and Feedback

Video #2 from the session: Bringing the Patient Voice to CNS Targeting Drug Development in DM. Dr. Gersham Dent, a neuroscientist working on the DM1 drug development program at Biogen, provides observations on panelists' CNS symptom experiences with regard to possible endpoint identification for future clinical trials of CNS-targeting therapies. From the 2017 MDF Annual Conference.

4/16/18 video

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