Ministerio de Ciencia e Innovación

#Específica ER# Duchenne Muscular Dystrophy Research Program (DMDRP)-USA

Duchenne Muscular Dystrophy Research Program (DMDRP)

Call: The goal of the FY19 DMDRP is to preserve and improve the function and quality of life and to extend the life span of all individuals with Duchenne. As such, the DMDRP is seeking to better support discovery and development of therapeutics, devices, and other interventions and to promote their rigorous clinical testing. Additionally, DMDRP supports the efforts of the National Institutes of Health (NIH) Muscular Dystrophy Coordinating Committee (MDCC) and the 2015 MDCC Action Plan for the Muscular Dystrophies, which prioritizes the needs to improve treatments and reduce the disease burden for muscular dystrophy including Duchenne.

All applications for the FY19 DMDRP Idea Development Award must address opportunities and challenges in the development of safe and effective macromolecular and cellular therapies that address primary pathology of DMD. Eligible therapeutic strategies include:

  • Gene therapy, genome editing, oligonucleotide therapies, exon skipping, protein therapeutics, and cell therapies. Studies proposed under this award may include:
  • Delivery to skeletal muscle and heart (e.g., ligand assisted, nanoparticles, identification of biological barriers to delivery, and alternative vectors)
  • Immunosuppression, vector modification, and other strategies to facilitate repeat administration of biologic therapies
  • Targeting muscle stem cells
  • Cell-based therapies, including but not limited to: selection of novel cell types, expansion, cell delivery and homing, differentiation, and integration

Deadline: Pre-Application: August 21, 2019. Preproposal is required; application submission is by invitation only. Application:December 4, 2019

Detailed information

Entidad Financiadora: Department of Defense os USA

Plazo de Presentación: 21/08/2019

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