Building Strength Together Webinar Series
The AFBS “Building Strength Together” Webinar Series is a collection of informative and supportive webinars designed specifically for the Nemaline Myopathy (NM) community. We cover a wide range of topics relevant to individuals affected by NM, their families, caregivers, and healthcare providers. From expert discussions on the latest research and treatments to practical advice on managing daily life with NM, these webinars offer valuable insights, resources, and connection. Join us as we learn, share, and build strength together!
A User’s Guide to the Latest in Respiratory Therapy Devices
This webinar is a must-watch for individuals affected by Nemaline Myopathy (NM), their caregivers, and anyone interested in learning more about respiratory health management. Respiratory therapist Chris Burgess BS, RRT-NPS, RCP, with his extensive experience in home-based care for patients with complex medical needs, offers valuable insights into the following devices and their applications:
Portable Mechanical Ventilation: Learn about the latest advancements in portable ventilators, their benefits, and how they can improve quality of life.
Mechanical Insufflation/Exsufflation (I/E): Understand how I/E devices can assist with airway clearance and improve respiratory function.
Oscillating Secretion Mobilization: Discover the benefits of oscillating secretion mobilization techniques and how they can help manage secretions.
Amplifying the NM Community’s Voice Webinar Series
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).
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.
Tools Used to Study the Spectrum of NM
The webinar features three expert Nemaline Myopathy (NM) researchers from across North America who share thorough updates on models (or tools) that have been created for use in gene-specific NM research.
Developing NM Community Priorities
This webinar offers personalized discussions among peers and experts to better understand challenges and opportunities related to Nemaline Myopathy, identifying therapeutic priorities and ultimately advancing research.
The Road to Treatment: Preparing the NM Community for Clinical Trials
This webinar will help you discover how YOU can shape the future of Nemaline Myopathy treatments. Learn about your vital role in engaging policymakers, navigating the FDA process, and accelerating treatment timelines. Drawing from her extraordinary experiences, guest speaker Jill Anne Castle, a trailblazer from the earliest DMD clinical trials to approval of a groundbreaking gene therapy, will empower us with insights into advancing research. Join esteemed CMD leaders, Rachel Alvarez and Rob Sunris, who’ll share invaluable strategies for engaging policy makers and navigating FDA interactions.