At A Foundation Building Strength, we’re driven in our unwavering pursuit of treatments for Nemaline Myopathy (NM). Today, we’re excited to announce a significant step forward in that journey: a new series of grants designed to accelerate the development of effective therapies for this rare neuromuscular disorder. This RFA represents a major opportunity for researchers to secure Nemaline Myopathy research grants and contribute to groundbreaking advancements in the field.
Investing in Innovation: Overview of the RFA
These grants represent a major investment in promising research, with the potential to transform the lives of individuals and families affected by NM. We’re committing significant resources, awarding up to five grants of $100,000 per year for up to three years, to fuel innovative projects that align with key areas identified by our Scientific Advisory Board:
- Small Molecule Research & Drug Screening: Identifying and validating small molecule therapies that can alleviate symptoms or modify disease progression. This includes high-throughput screening of existing and novel compounds, and preclinical development of promising candidates.
- Gene-Targeted Therapies: Developing genetic interventions to correct or compensate for NM-causing mutations. This includes research to improve gene delivery and expression for enhanced therapeutic efficacy.
- Bulbar Weakness & Respiratory Function: Investigating NM-associated bulbar dysfunction (affecting speech and swallowing) and developing interventions to improve respiratory function. This encompasses therapeutics, devices, and rehabilitation strategies.
- Supportive Care Devices & Functional Outcomes: Creating devices that enhance mobility, respiratory support, and daily functional abilities for NM patients. This includes exploring assistive technologies and developing reliable outcome measures to assess treatment effectiveness.
The Power of Collaboration: Our Scientific Advisory Board
These grants wouldn’t be possible without the guidance of our esteemed Scientific Advisory Board. This group of leading scientists, clinicians, and researchers provides invaluable expertise to help AFBS chart the most promising course towards treatments for NM.
AFBS Scientific Advisory Board Members:
- Dr. Alan Beggs, Co-Chair | Boston Children’s Hospital & Harvard University
- Dr. Gina Ravenscroft, Co-Chair | Harry Perkins Institute of Medical Research
- Dr. Lindsay Alfano | Nationwide Children’s Hospital & Ohio State University
- Dr. A. Reghan Foley | Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH
- Adrian Gomez | Ramboll, Parent Advocate
- Lucinda Lee | Patient Advocate
- Dr. Coen Ottenheijm | Amsterdam UMC & University of Arizona
- Wayne Sackett | Estuary Biotherapeutics
- Dr. Nicol Voermans | Radboud University Medical Center
Driving Progress: The Potential for Breakthroughs
With these Nemaline Myopathy research grants, we aim to fuel groundbreaking research that can lead to tangible improvements in the lives of those affected by NM. We believe that by supporting innovative projects and fostering collaboration, we can accelerate the development of effective treatments and bring hope and improved care to the NM community.
Join Us in this Vital Mission
We invite researchers with bold ideas and a passion for advancing NM research to submit their proposals. Together, we can drive progress and make a lasting impact on the lives of individuals and families affected by Nemaline Myopathy. We encourage all eligible researchers to apply for these Nemaline Myopathy research grants and contribute to this important cause.
For complete RFA details and application instructions, please visit our Request for Applications page on the AFBS website.
Donate: Your gift will support critical research and provide vital resources for NM families.
Stay Connected: Sign up for our email list to receive the latest updates from AFBS and learn more about NM research. Are you an individual or a caregiver of an individual affected by NM? Fill out the AFBS Contact Survey so we can connect you with valuable resources, support, and opportunities to get involved with upcoming research studies!