This competitive program aims to fund innovative and novel research proposals that seek to advance the understanding and management of 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

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