The International Gaucher Alliance and Kantar Health Launch the GARDIAN Registry to Accelerate the Understanding of Neuronopathic Gaucher Disease
Gaucher Disease (GD) is an autosomal recessive disease and the most common Lysosomal Storage Disorder, with an incidence of about 1 in 100,000 live births. Three types exist: type I, the most common, and types II and III, also called neuronopathic GD (nGD). Type II (GD2) is characterized by rapidly progressive neurological symptoms in babies; whereas type III (GD3) appears later in childhood and is defined by slower progression of neurological symptoms. Today, GD has no cure; treatment options are available to address some aspects, but none are specific to the neurological impairments of nGD.
GARDIAN is a global registry focused on patients with nGD. Through GARDIAN, the IGA aims to better understand the natural history of the disease and its impact on both patients and caregivers. As part of GARDIAN, disease specific patient-reported and observer-reported outcome scales will be developed.
Registry results will provide a comprehensive understanding of nGD which is essential to accelerate the early diagnosis and care for patients and ultimately improving patient outcomes. Real world evidence generated via GARDIAN will support clinical trial development and inform regulatory decision making.
“We’re excited to launch GARDIAN and wish to show our gratitude of our donors that are generously supporting this endeavor,” said
“We are delighted to partner with the IGA and our donors in an effort to help better understand neuronopathic Gaucher Disease and bring new therapies quicker to patients,” said
ABOUT THE INTERNATIONAL GAUCHER ALLIANCE (IGA)
The IGA is an international umbrella group representing the interests of Gaucher patients and those of not-for-profit Gaucher patient groups as well as rare disease groups throughout the world. While the IGA has an office in