Rare Disease Report

Chapter 1: Introduction

TITLE: Rare Diseases Resource Guide, 4th Edition
Subtitle: A Guide for Clinicians Managing Patients with Rare Conditions

Table of Contents
Chapter 1. Introduction
  • What Is a Rare Disease?
  • Orphan Drug Act
  • Incentives for Drug Development
  • Has the Orphan Drug Act Been a Success?

Chapter 2. Diagnosing a Rare Disease
  • Delayed Diagnosis Is Common
  • Resources

Chapter 3. Treatment
  • Medication
  • Lifestyle Changes
  • Surgery
  • Treatment vs Cure
  • Long-Term Effects of Treatment

Chapter 4. Patient Education
  • The Internet and Medical Information
  • Recommended Websites for Patients
  • Websites to Avoid
  • Caring for the Caregiver

Chapter 5. Patient Finances
  • Insurance Issues
  • Pharmaceutical Company Assistance Programs
  • Patient Advocacy Groups
  • Raising Money for Financial Support
  • Useful Websites for Financial Assistance Programs

Chapter 6. Familial Chylomicronemia Syndrome
  • Pathophysiology
  • Diagnosis
  • Management
  • Resources

Chapter 7. Huntington’s Disease
  • Pathophysiology
  • Diagnosis
  • Management
  • Resources

Chapter 8. Polycythemia Vera
  • Pathophysiology
  • Diagnosis
  • Management
  • Resources

Chapter 9. Pompe Disease
  • Pathophysiology
  • Diagnosis
  • Management
  • Resources

Chapter 10. Periodic Paralysis
  • Pathophysiology
  • Diagnosis
  • Management
  • Resources

Chapter 11. Gaucher Disease
  • Pathophysiology
  • Diagnosis
  • Management
  • Resources

Chapter 12. Phenylketonuria
  • Pathophysiology
  • Diagnosis
  • Management
  • Resources

Chapter 13. Congenital Plasminogen Deficiency
 
  • Pathophysiology
  • Diagnosis
  • Management
  • Resources


Appendix A. Approved Orphan Drugs

Appendix B. Approved Patient Advocacy Groups


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