THE INTERNATIONAL GAUCHER ALLIANCE AND CERNER ENVIZA ENROLL FIRST PATIENT IN GARDIAN REGISTRY
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; type II and III also called neuronopathic GD (nGD). Today, GD has no cure; treatment options are available to address some aspects, but none are specific to the neurological impairments of nGD.
The GARDIAN registry will collect retrospective and prospective clinical and health outcome data for patients diagnosed with Type 2 and 3 Gaucher disease. Caregivers will provide their perspective for pediatric and some adult patients, as well as the impact of caring for a family member with nGD. Patients and caregivers will complete new patient and caregiver reported outcomes instruments, which are the basis of the first nGD specific instruments available.
"There remains huge unmet needs in care, support, understanding and effective treatments for the neurological manifestations of the disease. GARDIAN will play an important role into better understanding the impact of the disease on patients and caregivers and ultimately enabling improved quality of life and better patient outcomes," said
Data collection will be made through Atom5™, Aparito's iOS & Android-compatible web and mobile app platform, which can capture eConsent, multiple, high-frequency data points from video, voice, wearables and electronic patient reported outcomes (ePROs). The Atom5™ global platform operates under ISO143485 QMS and ISO/IEC 27001 ISMS accreditations and is FDA CFR21 Part 11 compliant.
"We are excited to be part of this important patient-driven initiative, and that our Atom5™ platform will play a central part in generating insightful data which can support the whole community," said Dr.
The GARDIAN registry aims to provide a more comprehensive understanding of the natural history of disease for nGD patients in order to accelerate early diagnosis and care for patients. Real-world evidence derived from GARDIAN will support compound development and inform the feasibility, protocol design and implementation of clinical trials.
"We are delighted to achieve this important milestone and look forward to seeing the GARDIAN registry grow," said
ABOUT THE INTERNATIONAL GAUCHER ALLIANCE (IGA)
The IGA is an international umbrella group representing the interest of Gaucher patients and those of non-for-profit Gaucher patient groups as well as rare disease groups throughout the world. While the IGA has an office in
Aparito is a global health tech company that brings clinical trials to patients and unlocks real-world data through mobile apps, video assessments and wearable devices. We provide innovative patient-centric solutions that integrate required clinical and regulatory expertise to capture patient data and develop digital endpoints for hybrid and decentralised clinical trials.
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IGA : Harry Albright firstname.lastname@example.org; Cerner: Stephanie Greenwood, email@example.com; Aparito : Alexander Guest firstname.lastname@example.org or email@example.com