Amplifying the NM Community’s Voice” Webinar Recap

In our recent webinar, we delved into how you can play a pivotal role in shaping the future of Nemaline Myopathy treatments, underscoring the essential role the community plays in accelerating treatment timelines. We were honored to be joined by exceptional guest speakers, including Jill Anne Castle, a true pioneer in the field with firsthand experience in DMD clinical trials and the approval of groundbreaking gene therapies. Additionally, esteemed leaders Rachel Alvarez and Rob Sunris, representing Cure CMD, shared invaluable strategies for patient led research progress. Let’s take a closer look at the highlights.

New Resources for the Nemaline Myopathy Community

Founder of A Foundation Building Strength (AFBS), Marc Guillet, began the webinar by sharing a range of new resources tailored for the Nemaline Myopathy community. He highlighted our new website,, a comprehensive hub for all things related to Nemaline Myopathy. Here, you’ll discover an array of materials designed to deepen your understanding of Nemaline Myopathy and facilitate connections within the community. Whether you wish to support our efforts through donations, delve into our ongoing research, or actively contribute to the advancement of research, there are numerous avenues for you to make a meaningful difference. Additionally, Marc introduced two invaluable resources: the NM Resource Kit and the NM Awareness Card, which you can explore further in our dedicated blog post.

Sharing Your Story: The Power of Community Spotlight

Marc emphasized the importance of community members sharing their personal stories. AFBS’s Community Spotlight Series is designed to recognize and celebrate the unique individuals within the NM community. The Community Spotlights offer a glimpse into the lives of those living with NM, highlighting the rich diversity they represent. If you’re interested in sharing your own story and being featured on the AFBS website, we encourage you to fill out our questionnaire.

Insights from Our Esteemed Guest Speakers

Our guest speaker, Jill Anne Castle, M.Ed, a dedicated caregiver of a DMD-affected individual, offered invaluable insights drawn from her remarkable journey through clinical trials. From her involvement in the very first DMD clinical trial to bearing witness to the FDA’s approval of a groundbreaking gene therapy for DMD, her experiences shed light on the extraordinary impact of patient involvement.

Rachel Alvarez, the Executive Director of Cure CMD, played a pivotal role in organizing their 2022 Externally-Led Patient Focused Drug Development meeting with the FDA. This event also led to the creation of the inspiring film, “The Tenacity of Hope.” Rachel shared insights into the paramount importance of the patient’s voice and the significance of sharing one’s personal story.

Rob Sunris, President of Cure CMD and a parent of a CMD-affected individual, echoed Rachel’s message and shared his own experiences, feedback, and invaluable lessons learned from their FDA meeting and the creation of the aforementioned film.

You can watch the full film, “Tenacity of Hope” created by Cure CMD for their FDA meeting

We also hear from Jennifer McNary, a rare disease caregiver, speaking in a FDA patient-focused drug development listening meeting, which can be viewed here.

Dive Deeper with Q&A

Following these engaging presentations, a lively Q&A session ensued. Participants posed pressing questions, including inquiries about AFBS’s clinical and preclinical work, the timeline for upcoming clinical trials, and the best way to contribute to Natural History collection. All these queries have been addressed in the recorded webinar.

What’s Next: Your Action Plan

As you move forward on your journey within the Nemaline Myopathy community, we invite you to explore our new patient resources, including the NM Resource Kit and NM Awareness Card. Should you desire NM Awareness Cards, you can order them here.

We also encourage you to add your voice to the collective by sharing your personal story with AFBS. Simply complete our questionnaire to join the Community Spotlight Series and contribute to our growing tapestry of experiences.

Stay informed about our progress and upcoming events by signing up for our newsletter.

Mark your calendars for our next webinar scheduled for November 11th, 2023, at 8 am PT/11 am ET. Together, we’ll continue our journey down the road to progress to discover treatments for Nemaline Myopathy.

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