All USA USA Territories: American Samoa (USA); Guam (USA); Puerto Rico (USA); Virgin Islands (USA); Northern Mariana Islands (USA); USA Compact Free Associations:The Federated States of Micronesia (USA) Marshall Islands (USA) Republic of Palau (USA) International, Israel and Canada.
Grants to USA, Canada, and International researchers associated with institutions for research projects related to Duchenne muscular dystrophy. Funding is intended to support studies that involve basic and clinical research of gene therapy for DMD. The purpose of the program is to advance the science, treatment, and assessment of DMD.
Duchenne muscular dystrophy (DMD)
Area of Interest for this RFP:
Research projects that will be considered for support include the following basic and clinical research of gene therapy for DMD:
What is the prevalence of Adeno-associated virus (AAV) neutralizing antibodies within a DMD patient population
Strategies that can circumvent pre-existing neutralizing antibodies (nABs) to AAV that may allow future re-dosing or enabling eligibility for gene therapy for those patients with pre-existing nABs
What mechanism underpin the immunologic adverse effects of gene therapy and strategies that can be used to mitigate the risk and treat adverse responses
What mechanisms influence the durability of response to gene therapy and what factors predict response to guide decision making
Innovative and novel approaches that can support assessment and long-term follow-up for those DMD boys receiving gene therapy
Establish Minimal Clinically Important Difference (MCID) for relevant assessment used in DMD
Identify novel risk factors and potential management strategies for rapid DMD disease progression, cognitive impairment or development of other co-morbidities
Understand and predict response to treatment and the disease course of DMD
Develop and/or validate diagnostic, prognostic or response biomarkers in DMD
Understanding the clinical, economic and societal burden of DMD
How changes in motor function relate to changes in DMD patient and caregiver quality of life and/or activities of daily living
Treatment patterns in DMD, including the evolving use of steroids, exon-skippers, and gene therapy
Applicant Eligibility Criteria
• Only organizations are eligible to receive grants, not individuals or medical practice groups.
• The applicant (PI) must have a medical or postdoctoral degree (MD, PhD, or equivalent), an advanced nursing degree (BSN with a MS/PhD), or a degree in Pharmacy, Physiotherapy, or Social Work.
• Applicant must be affiliated with a host institution.
• Both early career and experienced investigators are encouraged to apply and consideration will be given to all proposals meeting the selection criteria.
• RFP Release Date: 4/27/2023
• Full Proposal Due Date: 7/10/2023
• Review of Full Proposals by ERP: August 2023
• Anticipated Full Proposal Notification Date: September 2023
• Grants will be distributed following a fully executed agreement and submission of Final Protocol, documentation of IRB/IEC approval, regulatory approval (if applicable), exemption or waiver.
Number of Grants
Estimated Size of Grant
Individual projects requesting up to $100,000 will be considered.