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Peroxisomal Disorders (Peroxisomal Disorder) Summary

Description: A heterogeneous group of inherited metabolic disorders marked by absent or dysfunctional PEROXISOMES. Peroxisomal enzymatic abnormalities may be single or multiple. Biosynthetic peroxisomal pathways are compromised, including the ability to synthesize ether lipids and to oxidize long-chain fatty acid precursors. Diseases in this category include ZELLWEGER SYNDROME; INFANTILE REFSUM DISEASE; rhizomelic chondrodysplasia (CHONDRODYSPLASIA PUNCTATA, RHIZOMELIC); hyperpipecolic acidemia; neonatal adrenoleukodystrophy; and ADRENOLEUKODYSTROPHY (X-linked). Neurologic dysfunction is a prominent feature of most peroxisomal disorders.

Also Known As: Peroxisomal Disorder; Adrenoleukodystrophy, Autosomal, Neonatal Form; Peroxisomal Dysfunction, General; Peroxisomal Dysfunction, Multiple; Peroxisomal Dysfunction, Single; Acidemia, Hyperpipecolic; Acidemias, Hyperpipecolic; Adrenoleukodystrophies, Neonatal; Dysfunction, General Peroxisomal; Dysfunction, Multiple Peroxisomal; Dysfunction, Single Peroxisomal; Dysfunctions, General Peroxisomal; Dysfunctions, Multiple Peroxisomal; Dysfunctions, Single Peroxisomal; General Peroxisomal Dysfunction; General Peroxisomal Dysfunctions; Hyperpipecolic Acidemias; Multiple Peroxisomal Dysfunction; Multiple Peroxisomal Dysfunctions; Neonatal Adrenoleukodystrophies; Neonatal Adrenoleukodystrophy; Peroxisomal Dysfunctions, General; Peroxisomal Dysfunctions, Multiple; Peroxisomal Dysfunctions, Single; Single Peroxisomal Dysfunction; Single Peroxisomal Dysfunctions; Adrenoleukodystrophy, Neonatal; Hyperpipecolic Acidemia

Networked: 360 relevant articles (2 outcomes, 17 trials/studies) for this Disease

Key Drugs and Agents for Peroxisomal Disorders

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. Fatty Acids (Saturated Fatty Acids) : 1 outcome 4 studies in 96 results : IBA
  2. alkylglycerone-phosphate synthase : 1 outcome in 5 results : IBA
  3. Phytanic Acid : 3 studies in 46 results : IBA
  4. pristanic acid : 2 studies in 19 results : IBA
  5. Enzymes : 2 studies in 18 results : IBA
  6. pipecolic acid : 2 studies in 17 results : IBA
  7. Catalase : 2 studies in 12 results : IBA
  8. Immune Sera (Antisera) : 2 studies in 2 results : IBA
  9. Acyl-CoA Oxidase : 1 study in 11 results : IBA
  10. Lipids : 1 study in 10 results : IBA
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Diseases Related to Peroxisomal Disorders

  1. Zellweger Syndrome (Zellweger's Syndrome)
  2. Multiple Sulfatase Deficiency Disease (Mucosulfatidosis)
  3. Ichthyosis (Xeroderma)
  4. Adrenoleukodystrophy (Adrenoleukodystrophy, X-Linked)
  5. Infantile Refsum Disease (Infantile Phytanic Acid Storage Disease)
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Key Therapies for Peroxisomal Disorders

Efficacy Chart >>
  1. Ketogenic Diet : 1 result
  2. Cell Transplantation : 1 result
  3. Bone Marrow Transplantation (Transplantation, Bone Marrow) : 1 result
  4. Drug Therapy (Chemotherapy) : 1 result

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