WP1 DM disease mechanisms & innovative model systems. Proof of therapeutic concepts: target-to- hit
WP Lead: RUMC
Objectives:
This work package aims to advance understanding of Myotonic Dystrophy (DM) through comprehensive genetic characterization and innovative disease modeling. Objectives include establishing protocols to identify and characterize DM mutations, creating a panel of cis modifiers to explore genetic variability, and developing advanced "muscle-on-chip" models alongside neuromuscular junction (NMJ) and brain organoids for accurate representation of disease mechanisms. Additionally, it will provide insights into the dynamics of RNA foci formation and investigate the role of microRNAs as intermediates in DM pathology.
WP2 Design of effective ASO-based therapeutic strategies: hit-to-lead
WP Lead: UVEG
Objectives:
This work package focuses on advancing the development and effectiveness of Antisense Oligonucleotides (ASO) for therapeutic use in neuromuscular disorders. Objectives include evaluating the impact of ASOs on neural network function, designing and synthesizing novel ASO molecules, and identifying chemical modifications that enhance their therapeutic efficacy. Additionally, the project aims to discover and implement new strategies to significantly improve ASO internalization into target tissues, thereby optimizing their therapeutic potential.
WP3 Biomarkers of disease progression and therapeutic response: clinical trial readiness
WP Lead: INSERM
Objectives:
This work package aims to improve clinical understanding and patient management in Myotonic Dystrophy (DM) by identifying reliable biomarkers and strengthening patient engagement. Key objectives include compiling comprehensive lists of muscle and central nervous system (CNS) biomarkers for DM1, as well as biomarkers specific to DM2. The project also focuses on defining relevant clinical outcomes and fostering active clinical engagement with patients to support future diagnostic and therapeutic efforts.
WP4 Training and career development
WP Lead: UVEG
Objectives:
The objectives of this work package include the creation of personalized Career Development Plans for each Doctoral Candidate , the compilation of a comprehensive Final Report detailing all secondments undertaken by the candidate , and the preparation of reports on the transferable skills acquired through both network-wide and local training activities. The goal is to support the professional growth of the candidates and ensure thorough documentation of their training and mobility experiences.
WP5 Dissemination, exploitation, outreach and communication
WP Lead: INSERM
Objectives:
This work package focuses on the visibility, communication, and long-term impact of the project. It includes the launch and maintenance of the project website , the development of a comprehensive dissemination and exploitation plan outlining communication activities at both early and final stages of the project, and the delivery of a mid-term report assessing communication efforts and public engagement . Additionally, an in-depth inventory analysis of the EU biogas sector will be conducted, providing insights and future perspectives for the industry .
WP6 Management, recruitment and sustainability
WP Lead: INSERM
The focus of this work package is on project management, quality assurance, and long-term sustainability. Tasks include the development of key guiding documents such as the Project Handbook, Quality Assurance Plan, and the ENTRY-DM MSCA Green Charter , as well as regular monitoring through a Progress Report . The package also addresses the sustainability of the doctoral training program , the coordination and oversight provided by the Supervisory Board , and the establishment of a comprehensive Data Management Plan to ensure responsible handling of research data .
WP7 Ethics requirements
WP Lead: INSERM
Objectives:
This work package sets out the 'ethics requirements' that the project must comply with.
