Limb Girdle (LGMD) Core Dataset
TREAT-NMD have a LGMD Core dataset available for registries (clinically and patient reported) to collect. The LGMD dataset was published in November 2021 as our 1st LGMD dataset. There are no previous versions, this is an initial core dataset for all LGMD disease sub-types.
The dataset is for all registries collecting data on individuals with LGMD (clinician/patient/dual reported). However, not all items are mandatory for all registries. A mandatory data item in the core dataset means that designated registries must include it in their data collection forms and be able to store and provide the data, even if the data is not always available for all individuals. Both versions of the DMD dataset can be found in the dropdowns below.
Version 1 of the LGMD dataset has:
- 82 unique data items
- 80 mandatory items for clinically reported registries
- 57 mandatory items for patient reported registries
- 15 defined categories
- Examples, data rules & tools
Version 1 of the LGMD core dataset can be found here: LGMD Core Dataset (wed based dataset specification)
No previous version
The acceleration of therapeutic development in LGMDs, the potential need for clinical trial development and ultimately the need for post market surveillance, required collaboration and consensus on a core data set for LGMD registries. The creation of Version 1 of the LGMD dataset is lead to more appropriate, harmonised, and reproducible data. This can be reliably used for the analysis of natural history data to support clinical trial design and improve patient recruitment to trials. TREAT-NMD, through its network of registries, stakeholders, and partners, is ideally placed to drive forward the development of a unified consensus on an LGMD Core Dataset, by working with Key opinion leaders, ensuring stakeholder collaboration, and encouraging partnership engagement.
The LGMD dataset was created by consensus with key opinion leaders, regulators, and industry representatives. It was piloted with 10 registries in a 4-month feasibility exercise and then the dataset updated based on registry feedback. It was then. Once approved by the PI Michela Guglieri & chairs of the TGDOC, v1 of the dataset was published 23rd November 2021. The dataset is also published Global Registries platform
If you have further questions about the LGMD dataset or any questions regarding the implementation, please contact:
The Registries team – registries@treat-nmd.com
Registry Team Members
Neil Bennett
Global Registries Manager
Neil is an experienced science communicator; in the neuromuscular disease area, he has worked at Act...
Annie Poll
Research Communications Lead, TREAT-NMD
Annie trained in several academic research laboratories at Newcastle University before taking on pos...
Farjana Ali
Datasets and Global Registries Platform Coordinator
Samantha Barker
Registry Network Administrator
Samantha (or Sammy) joined TREAT-NMD in July 2023. She has a background in administration and scienc...