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Familial Dysautonomia (Riley Day Syndrome) Summary

Description: An autosomal disorder of the peripheral and autonomic nervous systems limited to individuals of Ashkenazic Jewish descent. Clinical manifestations are present at birth and include diminished lacrimation, defective thermoregulation, orthostatic hypotension (HYPOTENSION, ORTHOSTATIC), fixed pupils, excessive SWEATING, loss of pain and temperature sensation, and absent reflexes. Pathologic features include reduced numbers of small diameter peripheral nerve fibers and autonomic ganglion neurons. (From Adams et al., Principles of Neurology, 6th ed, p1348; Nat Genet 1993;4(2):160-4)

Also Known As: Riley Day Syndrome; HSAN Type III; Hereditary-Sensory and Autonomic Neuropathy Type III; Dysautonomia, Familial; Dominant Hereditary Sensory Neuropathy, Type III Show All >>

Networked: 149 relevant articles (6 outcomes, 6 trials/studies) for this Disease

Key Drugs and Agents for Familial Dysautonomia

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. Methacholine Chloride (Methacholine) : 1 outcome in 5 results : FDA 1
  2. Tocotrienols : 1 outcome in 4 results : IBA
  3. Substance P : 1 outcome in 2 results : IBA
  4. Midazolam (Versed) : 1 outcome in 2 results : FDA 31 Generic
  5. Insulin-Like Growth Factor I (IGF-1) : 1 outcome in 1 result : IBA
  6. Nerve Growth Factor (NGF) : 2 studies in 7 results : IBA
  7. Messenger RNA (mRNA) : 1 study in 13 results : IBA
  8. Phosphotransferases (Kinase) : 1 study in 10 results : IBA
  9. Dopamine (Intropin) : 1 study in 4 results : FDA 36 Generic
  10. Dopa Decarboxylase : 1 study in 1 result : IBA
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Diseases Related to Familial Dysautonomia

  1. Ataxia (Dyssynergia)
  2. Vomiting
  3. Nausea
  4. Familial Dysautonomia (Riley Day Syndrome)
  5. Canavan Disease (Leukodystrophy, Spongiform)
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Key Therapies for Familial Dysautonomia

Efficacy Chart >>
  1. Gastrostomy : 1 outcome 1 study in 6 results
  2. Fundoplication : 1 outcome in 7 results
  3. Chest Wall Oscillation : 1 outcome in 1 result
  4. Autonomic Denervation : 1 study in 3 results
  5. Anesthesia : 5 results
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