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International GARDIAN Ltd is pleased to announce the release of data collected to early April 2024 as reported by the first 26 patients and parents/caregivers.

Owned by the IGA, GARDIAN is a global registry created to improve disease understanding, management and support for patients living with GD2 and GD3 (nGD).

It is a research platform to provide evidence-based data for advancing disease management, designing safer treatments and improving patient outcomes.

Summary of key results

  • GARDIAN has data on 26 patients (96% GD3 and 4% GD2).
  • Data were provided by patients (38.5%) and parents/caregivers (61.5%).
  • There were equal proportions of female (50%) and male (50%) patients.
  • Patients had a mean age of 2.4 years at diagnosis and 14.5 years at registry entry.
  • The mean number of signs/symptoms at diagnosis was 9.3.
  • Neurological symptoms at diagnosis included abnormal eye movements (38%), squint (31%) and swallowing difficulties (27%).
  • Non-neurological symptoms most frequently experienced at diagnosis included an enlarged spleen (81%), enlarged liver (69%) and low haemoglobin levels (65%).
  • Between diagnosis and registry entry, the prevalence of most non-neurological symptoms and signs decreased.
  • Conversely, neurological signs and symptoms increased between diagnosis and registry entry.
  • The most common medical treatments involved enzyme replacement therapy (ERT) (96%), calcium/vitamin D supplementation (58%),and antiepileptic drugs (35%).
  • Surgeries included tracheostomy (12%), percutaneous endoscopic gastrostomy (feeding tube) (12%), and partial splenectomy (8%).Patients frequently used medical devices such as glasses (31%), feet orthoses (15%), or a wheelchair (15%).
  • Patients or their caregivers used services such as case managers (38%), home care (23%) or adapted school/teaching (23%).
  • Overall, 27% of patients had at least one family member diagnosed with GD2 or GD3, and 35% had a family member diagnosed withParkinson’s Disease.
  • According to the Patient Health Questionnaire (PHQ-9), 38% of patients reported that nearly every day over the past 2 weeks, they felt bad about themselves or that they were a failure and had let themselves or their family down.
  • A total of 96% of patients lived with their parents/siblings and 4% with a partner/spouse.
  • Patients had achieved a secondary (40%) or university level education (Bachelor’s or Master’s equivalent) (12%).
  • Overall, 24% of patients were employed (either full-time, part-time, or as self-employed).

Results presentation

Obtain detailed results

Detailed results from the registry are available to qualified researchers and others in the field. For more details on what data is available and how to purchase it, please contact us.