Exciting Upgrade for JWMDRC Neuromuscular Patient Registries

In a significant step forward for the neuromuscular research community, the John Walton Muscular Dystrophy Research Centre (JWMDRC) has announced the transition of several of its patient registries to a new, state-of-the-art software platform, developed by Newcastle University. This move, supported by Muscular Dystrophy UK and our individual registry funders*, promises to enhance the user experience, improve data quality and completeness, and streamline resources for greater efficiency.

The national registries for FSHD, Myotonic Dystrophy, and SMA will be the first to migrate to the new platform, with some international registries to follow. The FKRP and COL6 international registries will transfer to a new version of our current system tailored to the LGMD disease area.  

The newly developed Newcastle University platform boasts a range of custom-built features tailored to the specific needs of the registries and offers numerous benefits to the neuromuscular research community, including:

• Improved customisation and flexibility: The easily adaptable infrastructure is designed to facilitate the creation of registries in new disease areas and support standalone studies, for example post-marketing surveillance projects and research surveys. This adaptability is crucial for responding to emerging research needs and opportunities in a timely manner, allowing the rare disease community to stay at the forefront of neuromuscular research. The ability to conduct these studies with greater speed and precision will support the development of new treatments and interventions, ultimately benefiting patients worldwide.

• Enhanced Efficiency and Data Quality: The transition will reduce data duplication, streamline resources, and improve data quality and completeness for both historical and new data across our registries. The new platform also offers improved data analysis capabilities, and powerful tools to extract meaningful insights from the data, driving forward the understanding and treatment of neuromuscular diseases. 

• Long-Term Sustainability: By increasing efficiencies in the areas of IT development and data management, the new platform enhances the long-term sustainability of the JWMDRC patient registries. Registry managers will have the ability to build and manage questionnaires directly, reducing timelines and associated costs.

• Strengthening Community Relationships: use of the improved platform will strengthen existing relationships with neuromuscular disease communities and increase participant numbers. The significantly increased range of data collected via the expanded registry datasets, alongside improved data quality will facilitate neuromuscular research both nationally and globally.

Several pharmaceutical companies have generously donated to fund the migration of existing data to the new platform; these multi-stakeholder contributions are crucial in achieving the project's main goals, and any further funding will enable additional enhancements to further improve the user experience.

Profs. Volker Straub and Chiara Marini-Bettolo, Principal Investigators of the registries, expressed their excitement about the transition:

"This new platform represents a major leap forward for our patient registries. The improved data quality and efficiency will greatly benefit our research efforts and, ultimately, the patients we serve. We are grateful for the support from our pharmaceutical industry partners and look forward to the positive impact this transition will bring. This project demonstrates just how impactful collaboration with different industry partners can be to the neuromuscular field.”

As the JWMDRC continues to accept donations for this vital project, the research community eagerly anticipates the numerous advancements and improvements that this new platform will bring, fostering a brighter future for neuromuscular research and patient care.

To learn more about how your company can join our growing number of supporters, please contact [email protected].

*Individual disease-specific patient registries migrating to the new platform are also supported by Myotonic Dystrophy Support Group, Cure DM, Adult SMA REACH, Myotubular Trust, MTM-CNM Family Connection, Astellas Pharma Inc, Roche, and Biogen.