Ultragenyx CDKL5 deficiency disorder study

Recruitment goal
30
participants
Launched in
2020
Status
Active
Image
Partner logo

Overview

About our partner: Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases, including CDKL5 deficiency disorder. Ultragenyx is currently in the early, pre-clinical stage of its CDKL5 deficiency disorder program and is interested in better understanding the clinical features, the natural history and the progression of the disease over time.

Study goal

Through this research, Ultragenyx aims to:

  • Understand the diagnosis and medical journeys of patients living with CDKL5 deficiency disorder
  • Learn about clinical testing, disease management and clinical milestones of CDKL5 deficiency disorder
  • Understand CDKL5 deficiency disorder symptoms, health complications and other disease-related conditions
  • Understand the use of medications and treatment plans among patients diagnosed with CDKL5 deficiency disorder
  • Learn what types of healthcare providers and specialists that patients visit, and the types of medical tests used to diagnose and monitor CDKL5 deficiency disorder

Research partners

About AllStripes

AllStripes is a platform that makes it easy for rare disease patients and caregivers to contribute to drug development research from home.

AllStripes improves the speed and design of drug trials by extracting and analyzing research-grade data from de-identified medical records, creating a pool of new knowledge about each rare condition.

Access your records, drive treatment research