Nemaline Myopathy Research Grants (RFA)

Nemaline Myopathy Research Grants

Funder: A Foundation Building Strength (AFBS)
Status: Most recent RFA cycle is closed as of the extraction date. Future cycles may be available.

Overview

AFBS launched a new series of research grants designed to accelerate the development of effective therapies for Nemaline Myopathy (NM), a rare neuromuscular disorder. The foundation commits significant resources to fund innovative projects aligned with key research areas identified by their Scientific Advisory Board.

Award Details

Amount: Up to $100,000 per year for up to three years Number of Awards: Up to five grants available Total Potential Funding: Up to $1.5 million across all grants

Focus Areas

The RFA targets research in the following key areas:

• Small Molecule Research & Drug Screening: Identifying and validating small molecule therapies to alleviate symptoms or modify disease progression, including 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, including research to improve gene delivery and expression
• Bulbar Weakness & Respiratory Function: Investigating NM-associated bulbar dysfunction (affecting speech and swallowing) and developing interventions to improve respiratory function through therapeutics, devices, and rehabilitation strategies
• Supportive Care Devices & Functional Outcomes: Creating devices that enhance mobility, respiratory support, and daily functional abilities for NM patients, including assistive technologies and reliable outcome measures

About Nemaline Myopathy

NM is a rare genetic neuromuscular disorder that causes muscle weakness and decreased muscle tone (hypotonia). Severity varies widely—from mild symptoms with minimal daily impact to severe disability and life-threatening complications.

Eligibility

Researchers at academic medical centers, universities, and relevant industry entities are encouraged to apply.

Scientific Guidance

Grants are guided by the AFBS Scientific Advisory Board, composed of leading scientists, clinicians, and researchers:
• 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

Contact Information

Scientific Director: Gustavo Dziewczapolski, PhD Email: gustavo@buildingstrength.org General Email: info@buildingstrength.org

Additional Notes

AFBS welcomes inquiries and concept discussions from investigators whose work aligns with strategic priorities. Prospective applicants are encouraged to contact the Scientific Director for further discussion before applying.