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Description

Ouverture d’une bourse de recherche (Request for Proposal (RFP)) afin de soutenir des initiatives destinées à faire progresser la compréhension et la prise en charge de la dystrophie musculaire de Duchenne (DMD).

Ce programme nommé «Global Awards for Research in DMD » est proposé par les équipes médicales de Pfizer.

Ce nouvel appel à projet porte sur les thématiques listées ci-dessous (en anglais) :

  • 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

Les financements pourront s’élever à 100 000 dollars par projet sélectionné.

Toutes les informations relatives à ce programme (domaines d’intérêt, délais et processus de soumission et d’approbation) sont détaillées dans le document en pièce jointe. Madame Amanda Stein est également à votre disposition pour toutes questions concernant le processus de soumission.

Financement