Site Map

Pages:

• Become Part of the Solution: Participate in Clinical Research
• Collecting Rare Disease Data: Why it Matters
• Conditions We Study
  • Angelman Syndrome (AS)
  • CDKL5 Deficiency Disorder (CDD)
  • Creatine Transporter Deficiency (CTD)
  • Duchenne Muscular Dystrophy (DMD)
  • Glycogen Storage Disease Type Ia (GSDIa)
  • Glycogen Storage Disease Type III (GSDIII)
  • Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
  • Mucopolysaccharidosis type VII (MPS VII)
  • Ornithine Transcarbamylase (OTC) Deficiency
  • Osteogenesis Imperfecta (OI)
  • Tumor-Induced Osteomalacia (TIO)
  • Wilson Disease (WD)
  • X-linked Hypophosphatemia (XLH)
• Contact Us
• Genetic Testing 101: Know Your Genes
• Home
• Rare & Prepared: Emergency Planning for Individuals with Complex Healthcare Needs
• Rare Experiences
• Rare Journey Resources
  • Getting Educated
  • Managing Daily Life
  • Patient Advocacy Groups
  • Preparing to Advocate
  • Searching for a Diagnosis
  • Taking Ownership
  • Transitioning Care
  • Understanding Diagnosis
• Site Map
• Spotlight on Rare Disease Caregivers
• Tell Us What You Think!
• Transitioning to Adulthood
• Ultragenyx Advocacy
  • Our Advocacy Team
• Ultragenyx Research
• Understanding Drug Development
  • About Clinical Trials
    • Supporting Access for All Patients
  • Understanding Gene Therapy
  • Understanding mRNA Therapy
• Your Voice Matters: How to Engage with the FDA