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A world without pain or tears

Felicia B Axelrod1

  • 1Dysautonomia Treatment and Evaluation Center, NYU Medical Center, 530 First Avenue, suite 9Q, New York, NY 10016, USA. felicia.axelrod@med.nyu.edu

Clinical Autonomic Research : Official Journal of the Clinical Autonomic Research Society
|May 10, 2006
PubMed

Insights

Familial dysautonomia (FD) is a genetic disorder impacting the nervous system. Advances in supportive care and genetic research offer improved treatments and prognosis for affected children.

Area of Science:

  • Genetics
  • Pediatrics
  • Neurology

Background:

  • Familial dysautonomia (FD) is a rare genetic disorder impacting the sensory and autonomic nervous system development in children.
  • Historically, FD has presented significant challenges in morbidity and mortality.
  • Recent advancements in genetic understanding offer new therapeutic avenues.

Purpose of the Study:

  • To review current supportive treatment modalities for familial dysautonomia.
  • To discuss the rationale behind existing treatments.
  • To explore innovative, genetically-informed treatment strategies for FD.

Main Methods:

  • Literature review of supportive care for FD.
  • Analysis of recent genetic research findings.
  • Synthesis of current and emerging treatment approaches.

Main Results:

  • Supportive treatments have significantly improved survival and quality of life for children with FD over the past 35 years.
  • Genetic breakthroughs are paving the way for novel therapeutic strategies.
  • New treatments aim to modify genetic expression and alter disease progression.

Conclusions:

  • Current supportive care is crucial for managing familial dysautonomia.
  • Emerging genetic therapies hold promise for fundamentally altering the prognosis of FD.
  • A combination of supportive and genetic-based treatments is key for future management.

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