Research Grant - Global Awards for Research in DMD-AWARD

Clinical Area

• 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