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Digital Academy

This webinar is essential for meeting attendees and will cover everything you need to know to make an impact on the FDA.

Patient Focused Drug Development (PFDD) meeting: This year at our annual conference MDF will host the first community meeting focused on the Food and Drug Administration (FDA). MDF community members will educate the FDA and other federal agency representatives about symptoms and disease issues that are most important to patients and families, and guide their thinking on DM drug review. The FDA wants to understand which benefits patients seek from therapies and what risks they might be willing to accept to gain specific benefits.

Community members discuss daily living strategies for motivating their adult children living with juvenile-onset DM1.

Please note: This is an audio recording only. To view the accompanying presentation, please click here.

Three community members living with DM1 discuss daily living strategies and ways to cope and manage the disease.

Note: this is an audio recording only. To see the slides that accompany this presentation, please click here.

Dr. Laurence Mignon, PhD, Director of Clinical Development at Isis Pharmaceuticals, gives an update on the status of the DMPKrx Phase I/II trial.

Dr. Richard Moxley of the University of Rochester addresses different endocrine problems that challenge patients with mytonic dystrophy types 1 & 2, how they can lead to symptoms and be associated with alterations in blood test measurements, known treatments to mitigate potential symptoms and future opportunities to develop better understanding of these endocrine problems.

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