Diagnostics and Therapeutics for Genetic Diseases

Table of Contents

• INTRODUCTION
  • STUDY GOAL AND OBJECTIVES
  • SCOPE AND FORMAT
  • CONTRIBUTIONS OF THE STUDY AND AUDIENCE
  • METHODOLOGY AND INFORMATION SOURCES
  • ABOUT THE AUTHOR
  • RELATED BCC REPORTS
  • BCC ONLINE SERVICES
  • DISCLAIMER
• SUMMARY
  • Summary Table: U.S. MARKET FOR THERAPEUTICS AND DIAGNOSTICS FOR GENETIC DISEASES, BY SEGMENT THROUGH 2010 ($ MILLIONS)
  • Summary Figure: U.S. MARKET FOR THERAPEUTICS AND DIAGNOSTICS FOR GENETIC DISEASES, BY SEGMENT, 2000-2010 ($ MILLIONS)
• OVERVIEW
  • INTRODUCTION TO MONOGENIC DISEASES
    • GENETIC TESTING
    • CARRIER SCREENING
    • PRENATAL DIAGNOSTIC TESTING
      • Ultrasound
      • Amniocentesis
      • Chorionic Villus Sampling (CVS)
      • Maternal Serum Alpha-Fetoprotein (MSAFP)
      • Triple or Quadruple Screen
      • Advanced Maternal Age
  • Table 1 MATERNAL AGE AND RISK OF CHROMOSOMAL ABNORMALITIES
    • Maternal Medical Problems
    • Genetic Counseling
    • PREIMPLANTATION DIAGNOSIS
    • NEWBORN SCREENING
  • Table 2 NEWBORN SCREENING RECOMMENDED BY THE AMERICAN COLLEGE OF MEDICAL GENETICS
  • Table 2 (CONTINUED)
  • Table 3 STATES WITH MANDATED NEWBORN SCREENING FOR ALL 29 OF THE RECOMMENDED ACMG DISORDERS, DECEMBER 2005
  • Table 4 STATES WITH MANDATED NEWBORN SCREENING FOR AT LEAST 20 OF THE RECOMMENDED ACMG DISORDERS, DECEMBER 2005
    • PRESYMPTOMATIC TESTING
    • CONFIRMATIVE DIAGNOSIS
  • Table 5 COMMON DNA-BASED GENE TESTS
    • SUCCESSES AND LIMITATIONS OF GENETIC TESTING
    • REGULATORY POLICIES
    • INSURANCE
• MARKET BY DISEASE SEGMENT
  • CYSTIC FIBROSIS
    • INTRODUCTION
    • GENETICS
      • Genetics (Continued)
  • Table 6 INCIDENCE OF COMMON MUTATIONS IN CYSTIC FIBROSIS IN THE UNITED STATES
    • SYMPTOMS
      • Mild to Moderate CF Lung Disease
      • Advanced/Severe CF Lung Disease
      • Pancreatic Insufficiency
      • Diabetes
      • Liver Damage
      • Fertility
      • Osteoporosis
    • DIAGNOSIS
    • TREATMENT OVERVIEW
      • Treatment Overview (Continued)
  • Table 7 U.S. MARKET FOR CYSTIC FIBROSIS THERAPEUTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 1 U.S. MARKET FOR CYSTIC FIBROSIS THERAPEUTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
    • DIAGNOSTICS FOR CYSTIC FIBROSIS
  • Table 8 U.S. MARKET FOR CYSTIC FIBROSIS DIAGNOSTICS TESTING BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 2 U.S. MARKET FOR CYSTIC FIBROSIS DIAGNOSTICS TESTING BY SEGMENT, 2000-2010 ($ MILLIONS)
    • PHARMACEUTICALS IN TREATMENT OF CYSTIC FIBROSIS
      • Bronchodilators
      • Dnase
  • Table 9 HEALTH-RELATED ECONOMIC END POINTS OF PULMOZYME REPORTED DURING A 24-WEEK STUDY
    • Pancreatic Enzyme Therapy
  • Table 10 MAJOR MANUFACTURERS OF PANCREATIC ENZYME THERAPIES
    • CORTICOSTEROIDS
    • ANTIBIOTICS
  • Table 11 U.S. MARKET FOR PHARMACEUTICALS IN TREATMENT OF CYSTIC FIBROSIS BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
    • The Market for Pharmaceuticals in the Treatment of CF
      • Bronchodilators
      • Pancreatic Enzyme Therapies
      • Corticosteroids
      • Antibiotics
    • TRANSPLANTS IN TREATMENT OF CYSTIC FIBROSIS
      • Transplant Timing
      • Lung Function
      • Market for Transplants in the Treatment of Cystic Fibrosis
  • Table 12 U.S. MARKET FOR TRANSPLANTS IN TREATMENT OF CYSTIC FIBROSIS, BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
    • FUTURE DIRECTIONS IN CF TREATMENT
      • Ion Channel Modulators
      • Curcumin
      • Living Donor Transplants
      • Gene Therapy
    • DUCHENNE AND BECKER MUSCULAR DYSTROPHY
      • INTRODUCTION
      • MUTATIONS
      • SYMPTOMS
      • DIAGNOSIS
      • TREATMENT
  • Table 13 U.S. MARKET FOR MUSCULAR DYSTROPHY THERAPEUTICS, BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 3 U.S. MARKET FOR MUSCULAR DYSTROPHY THERAPEUTICS, BY SEGMENT, 2000-2010 ($ MILLIONS)
    • DIAGNOSTICS FOR DMD/BMD
  • Table 14 U.S. MARKET FOR DMD/BMD DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 4 U.S. MARKET FOR DMD/BMD DIAGNOSTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
    • Pharmaceuticals for the Treatment of Muscular Dystropies
  • Table 15 U.S. MARKET FOR PHARMACEUTICALS IN TREATMENT OF DMD/BMD BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
    • Physiotherapy
    • Mechanical Ventilatory Assistance/Volume Ventilators
    • FUTURE DIRECTIONS IN DMD/BMD TREATMENT
    • HEMOPHILIAS
    • HEMOPHILIA A
      • Symptoms
      • Genetics
      • Diagnosis
      • Therapy
    • HEMOPHILIA B
      • Genetics
    • HEMOPHILIA C
    • VON WILLEBRAND DISEASE
    • HEMOPHILIA TREATMENT
  • Table 16 U.S. MARKET FOR HEMOPHILIA THERAPEUTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 5 U.S. MARKET FOR HEMOPHILIA THERAPEUTICS BY SEGMENT, 2000- 2010 ($ MILLIONS)
    • DIAGNOSTICS FOR HEMOPHILIAS
  • Table 17 U.S. MARKET FOR HEMOPHILIA DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 6 U.S. MARKET FOR HEMOPHILIA DIAGNOSTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
    • PHARMACEUTICALS IN TREATMENT OF HEMOPHILIAS
      • Replacement Therapy
  • Table 18 MAJOR CLOTTING FACTOR REPLACEMENT THERAPIES AND MANUFACTURERS IN THE U.S.
  • Table 18 (CONTINUED)
    • Other Pharmaceuticals
      • Desmopressin
      • Antifibrinolytic Drugs
    • Market for Pharmaceuticals in the Treatment of Hemophilias
  • Table 19 U.S. MARKET FOR PHARMACEUTICALS IN THE TREATMENT OF HEMOPHILIA BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
    • TRANSPLANTS IN TREATMENT OF HEMOPHILIAS
    • FUTURE DIRECTIONS
      • Improved Purification Processes
      • Gene Therapy
  • Table 20 GENE THERAPY TRIALS FOR HEMOPHILIA
    • HEREDITARY HEMOCHROMATOSIS
      • INTRODUCTION
      • GENETICS
      • SYMPTOMS
      • DIAGNOSIS
      • TREATMENT
  • Table 21 U.S. MARKET FOR HEREDITARY HEMACHROMATOSIS THERAPEUTICS, BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 7 U.S. MARKET FOR HEREDITARY HEMACHROMATOSIS THERAPEUTICS, BY SEGMENT, 2000-2010 ($ MILLIONS)
    • DIAGNOSTICS FOR HEREDITARY HEMACHROMATOSIS
  • Table 22 U.S. MARKET FOR HEREDITARY HEMACHROMATOSIS DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 8 U.S. MARKET FOR HEREDITARY HEMACHROMATOSIS BY CONFIRMATORY DIAGNOSIS, 2002-2010 ($ MILLIONS)
    • TRANSFUSIONS/ PHLEBOTOMY IN THE TREATMENT OF HEREDITARY HEMACHROMATOSIS
  • Table 23 U.S. MARKET FOR TRANSFUSIONS IN THE TREATMENT OF HEREDITARY HEMACHROMATOSIS BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
    • POLYCYSTIC KIDNEY DISEASE
      • INTRODUCTION
      • GENETICS
      • DIAGNOSIS
      • AUTOSOMAL DOMINANT PKD
        • Symptoms
        • Diagnosis
        • Treatment
          • Pain Relief
          • Urinary Tract Infections
          • High Blood Pressure
          • End-Stage Renal Disease (ESRD)
      • AUTOSOMAL RECESSIVE PKD
        • Genetics
        • Symptoms
        • Diagnosis
        • Treatment Overview
        • The U.S. Market for Polycystic Kidney Disease Therapeutics
  • Table 24 U.S. MARKET FOR POLYCYSTIC KIDNEY DISEASE THERAPEUTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 9 U.S. MARKET FOR POLYCYSTIC KIDNEY DISEASE THERAPEUTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
    • DIAGNOSTICS FOR POLYCYSTIC KIDNEY DISEASE
  • Table 25 U.S. MARKET FOR POLYCYSTIC KIDNEY DISEASE DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 10 U.S. MARKET FOR POLYCYSTIC KIDNEY DISEASE DIAGNOSTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
    • PHARMACEUTICALS IN TREATMENT OF POLYCYSTIC KIDNEY DISEASE
      • Vitamin D Analogues
      • Erythropoietin (EPO)
      • IV Iron Therapy
  • Table 26 U.S. MARKET FOR PHARMACEUTICALS IN THE TREATMENT OF POLYCYSTIC KIDNEY DISEASE BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
    • DIALYSIS IN TREATMENT OF POLYCYSTIC KIDNEY DISEASE
  • Table 27 U.S. MARKET FOR DIALYSIS IN TREATMENT OF POLYCYSTIC KIDNEY DISEASE BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
    • TRANSPLANTS IN TREATMENT OF POLYCYSTIC KIDNEY DISEASE
  • Table 28 U.S. MARKET FOR TRANSPLANTS IN TREATMENT OF POLYCYSTIC KIDNEY DISEASE, BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
    • SICKLE CELL AND RELATED ANEMIAS
      • INTRODUCTION
      • GENETICS
        • Genetics (Continued)
      • SYMPTOMS
        • Vasoocclusive Crises
        • Aplastic Crisis
        • Splenic Sequestration Crisis.
      • DIAGNOSIS
        • Newborn Screening
      • TREATMENT OVERVIEW
        • Febrile Illness
        • Painful (Vaso-Occlusive) Crises
        • Acute Chest Crisis
        • Hydroxyurea
        • Blood Transfusions
  • Table 29 U.S. MARKET FOR SICKLE CELL ANEMIA THERAPEUTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 11 U.S. MARKET FOR SICKLE CELL ANEMIA THERAPEUTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
    • DIAGNOSTICS FOR SICKLE CELL ANEMIA
  • Table 30 U.S. MARKET FOR SICKLE CELL ANEMIA DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 12 MARKET FOR SICKLE CELL ANEMIA DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
    • PHARMACEUTICALS IN TREATMENT OF SICKLE CELL AND RELATED ANEMIAS
      • Hydroxyurea
      • Emerging Pharamceuticals
        • Erythropoietin (EPO)
        • Clotrimazole
        • Fluocor
  • Table 31 U.S. MARKET FOR PHARMACEUTICALS IN THE TREATMENT OF SICKLE CELL ANEMIA BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
    • TRANSFUSIONS IN TREATMENT OF SICKLE CELL AND RELATED ANEMIAS
  • Table 32 U.S. MARKET FOR TRANSFUSIONS IN THE TREATMENT OF ANEMIAS BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
    • FUTURE TREATMENTS
    • THALASSEMIAS
      • INTRODUCTION
      • ALPHA THALASSEMIA
        • Genetics
        • Symptoms
      • BETA (b) THALASSEMIAS
        • Genetics
        • Symptoms
      • DELTA (d) THALASSEMIA
      • THALASSEMIA IN COMBINATION WITH OTHER HEMOGLOBINOPATHIES
      • DIAGNOSIS
      • TREATMENT OVERVIEW
  • Table 33 U.S. MARKET FOR THALASSEMIA THERAPEUTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 13 U.S. MARKET FOR THALASSEMIA THERAPEUTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
    • DIAGNOSTICS FOR THALASSEMIA
  • Table 34 U.S. MARKET FOR THALASSEMIA DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
  • Figure 14 U.S. MARKET FOR THALASSEMIA DIAGNOSTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
    • TRANSFUSIONS IN TREATMENT OF THALASSEMIA
  • Table 35 U.S. MARKET FOR TRANSFUSIONS IN THE TREATMENT OF THALASSEMIAS BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
    • FUTURE TREATMENTS
• EMERGING THERAPEUTIC TARGETS
  • ENZYMATIC DEFICIENCIES
    • ALPHA-1-ANTITRYPSIN DEFICIENCY
      • Symptoms
      • Genetics
      • Treatment
    • GAUCHER DISEASE
      • Symptoms
      • Diagnosis
      • Genetics
      • Treatment
    • PHENYLKETONURIA
      • Diagnosis
      • Treatment
  • MOTOR-NERVOUS SYSTEM DISEASES
    • AMYOTROPHIC LATERAL SCLEROSIS
      • Symptoms
      • Diagnosis
      • Genetics
      • Treatment
    • FUTURE DIRECTIONS
    • FRAGILE X SYNDROME
      • Genetics
      • Diagnosis
      • Treatment
    • CANAVAN DISEASE
      • Symptoms
      • Genetics
      • Treatment
    • NEUROFIBROMATOSIS TYPE 1
      • Genetics
      • Symptoms
      • Symptoms (Continued)
      • Diagnosis
      • Treatment
  • IMMUNE SYSTEM DISORDERS
    • ATAXIA TELANGIECTASIA
      • Genetics
      • Symptoms
      • Treatment
      • Diagnosis
  • METABOLIC DISORDERS
    • MAPLE SYRUP URINE DISEASE
      • Symptoms
      • Genetics
      • Treatment
    • NIEMANN-PICK DISEASE
      • Genetics
      • Treatment
    • TAY-SACHS DISEASE
      • Symptoms
      • Genetics
      • Treatment and Future Directions
    • MUCOPOLYSACCHARIDE DISEASE (HURLERS DISEASE, SANFILLIPO SYNDROME, HUNTERS SYNDROME)
  • Table 36 MUCOPOLYSACCHARIDE DISEASES
    • MPS-I H, Hurler Syndrome
      • Diagnosis
      • Treatment
    • MPS-II, Hunter Syndrome
      • Genetics
      • Symptoms
      • Diagnosis
      • Treatment
    • MPS-III, Sanfillipo Syndrome
      • Diagnosis
      • Treatment
      • Future Therapies
• MARKET BY PRODUCT TYPE
  • DIAGNOSTICS FOR GENETIC DISEASE
  • Table 37 U.S. MARKET FOR DIAGNOSTICS OF GENETIC DISEASE BY DISEASE TYPE, THROUGH 2010 ($ MILLIONS)
  • Figure 15 U.S. MARKET FOR DIAGNOSTICS OF GENETIC DISEASE BY DISEASE TYPE, 2000-2010 ($ MILLIONS)
    • PHARMACEUTICAL TREATMENTS OF GENETIC DISEASE
  • Table 38 U.S. MARKET FOR PHARMACEUTICAL TREATMENTS OF GENETIC DISEASE BY DISEASE TYPE, THROUGH 2010 ($ MILLIONS)
    • TRANSPLANTATION TREATMENT OF GENETIC DISEASE
  • Table 39 U.S. MARKET FOR TRANSPLANTATION TREATMENT OF GENETIC DISEASE BY DISEASE TYPE, THROUGH 2010 ($ MILLIONS)
    • TRANSFUSION TREATMENT OF GENETIC DISEASE
  • Table 40 U.S. MARKET FOR TRANSFUSION TREATMENT OF GENETIC DISEASE, BY DISEASE TYPE ($ MILLIONS)
    • TRANSPLANTATION TREATMENT OF�ECONTINUED)
• PATENT EVALUATION
  • Table 41 MONOGENIC DISEASE TREATMENTS, PATENTS BY DISEASE, FEBRUARY 2004-FEBRUARY 2006
  • Table 42 MONOGENIC DISEASE TREATMENTS, PATENTS BY ASSIGNEE, FEBRUARY 2004-FEBRUARY 2006
  • Table 42 (CONTINUED)
  • Table 43 MONOGENIC DISEASE TREATMENTS, PATENTS BY REGION, FEBRUARY 2004-FEBRUARY 2006
  • Table 44 MONOGENIC DISEASE TREATMENTS, PATENTS BY INSTITUTION TYPE, FEBRUARY 2004-FEBRUARY 2006
    • RECENT PATENTS OF SIGNIFICANCE
      • METHOD FOR UPREGULATION OF CF TRANSMEMBRANE MEMBRANE CONDUCTANCE REGULATORS
      • ENHANCERS OF CFTR CHLORIDE CHANNEL FUNCTION
      • POTENTIAL NUTRACEUTICAL TREATMENT FOR PKD
      • SCREENING METHOD FOR COMPOUNDS TO TREAT PKD
      • GLYCOSYLATED, LOW ANTIGENICITY LOW IMMUNOGENICITY FACTOR VIII
      • ANTIVIRAL AGENT FOR PREVENTION OF CELL SICKLING
• OUTLOOK
  • FUTURE DIRECTIONS
    • IDENTIFICATION OF MODIFIER GENES AND MUTATIONS
    • NON-INVASIVE PRENATAL TESTING
    • GENE THERAPY
    • STEM CELL THERAPY
  • CHALLENGES AND ETHICAL CONSIDERATIONS
    • INSURANCE COVERAGE
    • OVER-THE-COUNTER OPTIONS
    • PRENATAL TESTING
  • COMPANY PROFILES
    • AMGEN
    • ATHENA DIAGNOSTICS, INC.
    • CHIRON, INC
    • GENAERA CORP
    • GENENTECH INC.
    • GENZYME CORP.
    • LABORATORY CORPORATION OF AMERICA (LABCORP)
    • MAXYGEN
    • MILLENIUM PHARMACEUTICALS
    • MYRIAD GENETICS
    • ORCHID BIOSCIENCES
      • Orchid Biosciences (Continued)
    • PFIZER INC.
    • QUEST DIAGNOSTICS
    • ROCHE (F. HOFFMANN-LA ROCHE LTD)
    • SANOFI-AVENTIS
    • VERTEX PHARMACEUTICALS, INC
      • Vertex Pharmaceuticals, Inc (Continued)
    • WYETH-AYERST, INC.
• LIST OF TABLES
• Summary Table:
• Table 1 MATERNAL AGE AND RISK OF CHROMOSOMAL ABNORMALITIES
• Table 2 NEWBORN SCREENING RECOMMENDED BY THE AMERICAN COLLEGE OF MEDICAL GENETICS
• Table 3 STATES WITH MANDATED NEWBORN SCREENING FOR ALL 29 OF THE RECOMMENDED ACMG DISORDERS, DECEMBER 2005
• Table 4 STATES WITH MANDATED NEWBORN SCREENING FOR AT LEAST 20 OF THE RECOMMENDED ACMG DISORDERS, DECEMBER 2005
• Table 5 COMMON DNA-BASED GENE TESTS
• Table 6 INCIDENCE OF COMMON MUTATIONS IN CYSTIC FIBROSIS IN THE UNITED STATES
• Table 7 U.S. MARKET FOR CYSTIC FIBROSIS THERAPEUTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 8 U.S. MARKET FOR CYSTIC FIBROSIS DIAGNOSTICS TESTING BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 9 HEALTH-RELATED ECONOMIC END POINTS OF PULMOZYME REPORTED DURING A 24-WEEK STUDY
• Table 10 MAJOR MANUFACTURERS OF PANCREATIC ENZYME THERAPIES
• Table 11 U.S. MARKET FOR PHARMACEUTICALS IN TREATMENT OF CYSTIC FIBROSIS BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
• Table 12 U.S. MARKET FOR TRANSPLANTS IN TREATMENT OF CYSTIC FIBROSIS, BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
• Table 13 U.S. MARKET FOR MUSCULAR DYSTROPHY THERAPEUTICS, BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 14 U.S. MARKET FOR DMD/BMD DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 15 U.S. MARKET FOR PHARMACEUTICALS IN TREATMENT OF DMD/BMD BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
• Table 16 U.S. MARKET FOR HEMOPHILIA THERAPEUTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 17 U.S. MARKET FOR HEMOPHILIA DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 18 MAJOR CLOTTING FACTOR REPLACEMENT THERAPIES AND MANUFACTURERS IN THE U.S.
• Table 19 U.S. MARKET FOR PHARMACEUTICALS IN THE TREATMENT OF HEMOPHILIAS BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
• Table 20 GENE THERAPY TRIALS FOR HEMOPHILIA
• Table 21 U.S. MARKET FOR HEREDITARY HEMACHROMATOSIS THERAPEUTICS, BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 22 U.S. MARKET FOR HEREDITARY HEMACHROMATOSIS DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 23 U.S. MARKET FOR TRANSFUSIONS IN THE TREATMENT OF HEREDITARY HEMACHROMATOSIS BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
• Table 24 U.S. MARKET FOR POLYCYSTIC KIDNEY DISEASE THERAPEUTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 25 U.S. MARKET FOR POLYCYSTIC KIDNEY DISEASE DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 26 U.S. MARKET FOR PHARMACEUTICALS IN THE TREATMENT OF POLYCYSTIC KIDNEY DISEASE BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
• Table 27 U.S. MARKET FOR DIALYSIS IN TREATMENT OF POLYCYSTIC KIDNEY DISEASE BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
• Table 28 U.S. MARKET FOR TRANSPLANTS IN TREATMENT OF POLYCYSTIC KIDNEY DISEASE, BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
• Table 29 U.S. MARKET FOR SICKLE CELL ANEMIA THERAPEUTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 30 U.S. MARKET FOR SICKLE CELL ANEMIA DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 31 U.S. MARKET FOR PHARMACEUTICALS IN THE TREATMENT OF SICKLE CELL ANEMIA BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
• Table 32 U.S. MARKET FOR TRANSFUSIONS IN THE TREATMENT OF ANEMIAS BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
• Table 33 U.S. MARKET FOR THALASSEMIA THERAPEUTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 34 U.S. MARKET FOR THALASSEMIA DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Table 35 U.S. MARKET FOR TRANSFUSIONS IN THE TREATMENT OF THALASSEMIAS BY PRODUCT TYPE, THROUGH 2010 ($ MILLIONS)
• Table 36 MUCOPOLYSACCHARIDE DISEASES
• Table 37 U.S. MARKET FOR DIAGNOSTICS OF GENETIC DISEASE BY DISEASE TYPE, THROUGH 2010 ($ MILLIONS)
• Table 38 U.S. MARKET FOR PHARMACEUTICAL TREATMENTS OF GENETIC DISEASE BY DISEASE TYPE, THROUGH 2010 ($ MILLIONS)
• Table 39 U.S. MARKET FOR TRANSPLANTATION TREATMENT OF GENETIC DISEASE BY DISEASE TYPE, THROUGH 2010 ($ MILLIONS)
• Table 40 U.S. MARKET FOR TRANSFUSION TREATMENT OF GENETIC DISEASE, BY DISEASE TYPE ($ MILLIONS)
• Table 41 MONOGENIC DISEASE TREATMENTS, PATENTS BY DISEASE, FEBRUARY 2004-FEBRUARY 2006
• Table 42 MONOGENIC DISEASE TREATMENTS, PATENTS BY ASSIGNEE, FEBRUARY 2004-FEBRUARY 2006
• Table 43 MONOGENIC DISEASE TREATMENTS, PATENTS BY REGION FEBRUARY 2004-FEBRUARY 2006
• Table 44 MONOGENIC DISEASE TREATMENTS, PATENTS BY INSTITUTION TYPE, FEBRUARY 2004-FEBRUARY 2006
• LIST OF FIGURES
• Summary Figure:
• Figure 1 U.S. MARKET FOR CYSTIC FIBROSIS THERAPEUTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
• Figure 2 U.S. MARKET FOR CYSTIC FIBROSIS DIAGNOSTICS TESTING BY SEGMENT, 2000-2010 ($ MILLIONS)
• Figure 3 U.S. MARKET FOR MUSCULAR DYSTROPHY THERAPEUTICS, BY SEGMENT, 2000-2010 ($ MILLIONS)
• Figure 4 U.S. MARKET FOR DMD/BMD DIAGNOSTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
• Figure 5 U.S. MARKET FOR HEMOPHILIA THERAPEUTICS BY SEGMENT, 2000- 2010 ($ MILLIONS)
• Figure 6 U.S. MARKET FOR HEMOPHILIA DIAGNOSTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
• Figure 7 U.S. MARKET FOR HEREDITARY HEMACHROMATOSIS THERAPEUTICS, BY SEGMENT, 2000-2010 ($ MILLIONS)
• Figure 8 U.S. MARKET FOR HEREDITARY HEMACHROMATOSIS BY CONFIRMATORY DIAGNOSIS, 2002-2010 ($ MILLIONS)
• Figure 9 U.S. MARKET FOR POLYCYSTIC KIDNEY DISEASE THERAPEUTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
• Figure 10 U.S. MARKET FOR POLYCYSTIC KIDNEY DISEASE DIAGNOSTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
• Figure 11 U.S. MARKET FOR SICKLE CELL ANEMIA THERAPEUTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
• Figure 12 MARKET FOR SICKLE CELL ANEMIA DIAGNOSTICS BY SEGMENT, THROUGH 2010 ($ MILLIONS)
• Figure 13 U.S. MARKET FOR THALASSEMIA THERAPEUTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
• Figure 14 U.S. MARKET FOR THALASSEMIA DIAGNOSTICS BY SEGMENT, 2000-2010 ($ MILLIONS)
• Figure 15 U.S. MARKET FOR DIAGNOSTICS OF GENETIC DISEASE BY DISEASE TYPE, 2000-2010 ($ MILLIONS)