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Current Landscape of Treatments for Wolfram Syndrome

Damien Abreu1, Fumihiko Urano2

  • 1Department of Medicine, Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, St. Louis, MO 63110, USA; Medical Scientist Training Program, Washington University School of Medicine, St. Louis, MO 63110, USA.

Trends in Pharmacological Sciences
|August 18, 2019
PubMed

Insights

Wolfram syndrome, a rare genetic disorder, currently lacks treatments. This study outlines a vision for developing novel therapeutic strategies to cure this progressive neurodegenerative disease.

Area of Science:

  • Genetics and rare diseases
  • Neurodegenerative disorders
  • Endocrinology

Background:

  • Wolfram syndrome is a rare genetic disorder.
  • Characterized by diabetes mellitus, optic nerve atrophy, and neurodegeneration.
  • Symptoms range from mild to severe.

Purpose of the Study:

  • To summarize a vision for novel treatment strategies.
  • To outline a path towards a cure for Wolfram syndrome.

Main Methods:

  • Literature review and expert opinion synthesis.
  • Conceptual framework development for therapeutic strategies.

Main Results:

  • Identification of key challenges in Wolfram syndrome treatment.
  • Proposal of innovative therapeutic approaches.

Conclusions:

  • Urgent need for innovative therapeutics for Wolfram syndrome.
  • A clear vision for developing novel treatment strategies and achieving a cure is presented.
Keywords:
WFS1Wolfram syndromeendoplasmic reticulumgene therapyregenerative therapy

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