Request for Applications

Priority will be given to projects that align with the following key areas:

1. Small Molecule Research & Drug Screening
   • Identification and validation of small molecule therapies with the potential to alleviate NM-specific symptoms and/or prevent disease progression.
   • Medium to High-throughput screening of existing or novel compounds with  therapeutic potential for NM 
   • Preclinical development and optimization of promising small molecules.
   • Development and validation of biochemical, cellular and animal assay systems and systematic compound testing strategies to identify viable drug candidates.
2. Gene-Targeted Therapies
   • Development of genetic interventions aimed at correcting or compensating for NM-related genetic mutations.
   • Research aimed at improving gene delivery vectors and expression of therapeutic constructs of relevance to NM to enhance therapeutic efficacy and/or reduce immunogenicity.
3. Bulbar Weakness & Respiratory Function
   • Research targeting NM-associated bulbar dysfunction, including swallowing and speech deficits.
   • Interventions to improve respiratory function, including therapeutics, devices, and rehabilitation strategies for individuals with NM.
   • Development of biomarkers or outcome measures relevant to bulbar and respiratory function in NM.
4. Supportive Care Devices & Functional Outcomes
   • Development of devices that enhance mobility, respiratory support, and daily functional abilities for NM patients.
   • Exploration of technologies to improve quality of life, including assistive and rehabilitative devices.
   • Research into outcome measures and biomarkers to assess therapeutic effectiveness in NM.

Proposals outside these areas will be considered if they have a compelling rationale and a strong potential translational impact on NM treatment.

A two-page LOI is required by April 15 prior to submitting a full proposal. LOIs should include:

• Lay Summary: Briefly describe the project and its potential impact on NM therapeutic development in simple terms.
• Introduction: Provide background and significance of the research.
• Specific Aims: Clearly outline the research goals.
• Methodology: Briefly describe the research material & methods.
• Budget & Key Personnel: Briefly describe the overall budget you will request and the key personnel, with affiliations, involved in the proposed project.  

Abstracts

• Lay Abstract: 300 words summarizing the research in non-technical language.
• Scientific Abstract: 300 words outlining the study’s aims, significance, methodology, and expected outcomes.

Research Plan (Maximum 5 pages, excluding references & appendices)

1. Hypothesis & Specific Aims (1 page max)
   • Clearly state the hypothesis and research objectives.
2. Background & Significance (1 page max)
   • Describe the scientific rationale and potential impact on NM treatment.
3. Preliminary Data (1 page max)
   • Summarize prior work, including relevant publications and supporting data.
4. Research Design & Methods (2 pages max)
   • Detail experimental approach, timeline, and methodologies.
5. Success Criteria (1 page max)
   • Define milestones, evaluation methods, and statistical analyses.
6. References (1 page max)
7. Appendices (1 page max)
   • If needed, add an Appendix including more detailed preliminary results.

Applicants Must:

• Hold an M.D., Ph.D., or equivalent degree and either lead an independent research laboratory at an accredited scientific institution or be affiliated with a start-up, small pharmaceutical, or biotechnology company conducting relevant research.
• Assume administrative and financial responsibility for the grant.
• Have access to institutional resources necessary for successful project execution.
• There are no geographic restrictions.

• Funding is milestone-based and subject to annual progress review.
• Written progress reports and virtual check-ins required quarterly (1-year projects) or semiannually (multi-year projects).
• Clinical and animal studies require regulatory approvals before funding initiation.

• Letter of Intent Deadline: April 15
• Full Proposal Deadline: June 16
• Notifications of Selected Grants: Expected by August 2025
• Submit applications and inquiries to: gustavo@buildingstrength.org