Our webinar series, “Amplifying the NM Community’s Voice” is aimed to engage and build an effective and lasting partnership among our stakeholders to drive forward patient centered research initiatives for the NM community. These webinars are available thanks to the support of PCORI (Patient Centered Outcomes Research Institute).
Past Webinar Recordings
Introduction to Community Involvement
The webinar shares results from a significant NM community survey, offers insight from AFBS-funded researcher, Alan Beggs, on the latest research discoveries, and highlights how community involvement is a critical step in bringing NM research to clinical trials.
Research Fundamentals, Part 1
The webinar defines PCOR (Patient Centered Research Outcomes and review of the role of the CRPs (Community Research Partners), reviews existing infrastructure in place to support NM research and how families can get involved. Dr. Michael Lawlor discusses the importance of human tissue resources in biomedical research.
Research Fundamentals, Part 2
The webinar shares the Toolkit for Patient-Focused Therapy Development, encourages participation in the CMDIR registry, and shares the importance of tissue sample donations. It also discussed Translational Science as an essential part of the path to treatment for NM.
Pathway to a Natural History Study
The webinar focuses on the pathway to a Natural History Study for Nemaline Myopathy. Guest speakers included Leslie Hayes, MD, Pediatric Neurologist, Lindsay Alfano, PT, DPT, PCS and Research Partner, and Alan Beggs, PhD and Scientific Partner. For more information on data literacy and medical research, watch this new NORD video.
Why Genetic Confirmation Matters for Treatment
The webinar focuses on the importance of genetic confirmation. Dr. Carsten Bönnemann, a world renowned board-certified Pediatric Neurologist, presents the “Gene Therapy Toolbox”. Genetic counselor, Brianna Gross shares “a roadmap to confirming genetic subtype”.
Listening to the Adult NM community
The webinar offers the NM-affected adult community a space for communication, and share feedback and guidance to help improve our overall efforts as an organization. Every voice matters and we want to hear from the entire community to better represent the research priorities.