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• 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)
  • Homozygous Familial Hypercholesterolemia (HoFH)
  • 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)
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• Genetic Testing 101: Know Your Genes
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• Transitioning to Adulthood
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    • Supporting Access for All Patients
  • Understanding Gene Therapy
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• Your Voice Matters: How to Engage with the FDA