NEW YORK, April 6 Reportlinker.com announces that a new market research report is available in its catalogue:
New Developments in Treating Genetic Disorders: The Global Market
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THIS REPORT:
Discussion of treatment options, key products and new developments in therapies for such genetic disorders as cystic fibrosis, Gaucher disease, hemophilia, Huntington's disease, sickle-cell disease, and Tay-Sachs disease
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Analysis of the market, citing data for 2008, estimates for 2009, and projections, with 5-year compound annual growth rates (CAGRs), through 2014
Examination of the market in chromosomal disorders, such as Down, Klinefelter's, Prader-Willi, and Turner's syndromes
Patent evaluations and company profiles.
Chapter- 1: INTRODUCTION
STUDY GOALS AND OBJECTIVES 1
REASONS FOR DOING THE STUDY 1
SCOPE OF REPORT 1
METHODOLOGY 2
ANALYST CREDENTIALS 2
RELATED BCC STUDIES 3
BCC ONLINE SERVICES 3
DISCLAIMER 3
Chapter-2: SUMMARY
GENETIC DISORDER MARKETS: MAJOR MARKETS SUMMARY 4
SUMMARY TABLE A GLOBAL GENETIC DISORDER PRODUCT REVENUES BY MAJOR MARKET SEGMENT, THROUGH 2014 ($ MILLIONS) 5
SUMMARY FIGURE GLOBAL GENETIC DISORDER PRODUCT REVENUES, 2007-2014 ($ MILLIONS) 5
LEADING MANUFACTURERS 5
SUMMARY TABLE B TOTAL COMPANY SALES FOR GENETIC DISORDER TREATMENT, 2009 ($ MILLIONS) 6
Chapter-3: INDUSTRY OVERVIEW
INTRODUCTION 7
HISTORY OF GENETIC RESEARCH 7
OVERVIEW OF DNA AND GENES 7
THE HUMAN GENOME PROJECT 8
HAPMAP PROJECT 9
TERMINOLOGY 9
TABLE 1 GLOSSARY OF GENETIC TERMS 9
TABLE 1 (CONTINUED) 10
TABLE 1 (CONTINUED) 11
TABLE 1 (CONTINUED) 12
TABLE 1 (CONTINUED) 13
TABLE 1 (CONTINUED) 14
TABLE 1 (CONTINUED) 15
TABLE 1 (CONTINUED) 16
GENES AND FAMILY HISTORY 16
OVERVIEW OF GENETIC DISORDERS 16
GENE MUTATIONS 16
CAUSES OF GENE MUTATIONS 17
GENETIC TESTING AND SCREENING 18
TESTING POPULATION GROUPS (ETHNOGENETICS) 19
DIRECT TO CONSUMER GENETIC TESTING 19
U.S. DEMOGRAPHICS 20
TABLE 2 NUMBER OF BIRTHS IN THE U.S., 2006-2010 21
TABLE 3 POPULATION GROWTH AND BIRTH STATISTICS U.S., 2011-2020 (THOUSANDS) 21
FIGURE 1 POPULATION GROWTH AND BIRTH STATISTICS U.S. 2011-2020 (THOUSANDS) 22
INTERNATIONAL DEMOGRAPHICS 23
TABLE 4 ESTIMATED WORLD POPULATION AND ANNUAL GROWTH BY SELECTED AGE GROUP AND SELECTED YEARS, 2000 THROUGH 2030 (MILLIONS) 23
FIGURE 2 ESTIMATED WORLD POPULATION BY SELECTED AGE GROUP AND SELECTED YEARS, 2000-2030 (MILLIONS) 23
TABLE 5 WORLD POPULATION BY SELECTED COUNTRIES, THROUGH 2014 (MILLIONS) 24
FIGURE 3 WORLD POPULATION BY SELECTED COUNTRIES, 2007-2014 (MILLIONS) 25
INTERNATIONAL BIRTH RATE STATISTICS 25
TABLE 6 BIRTHS FOR SELECTED COUNTRIES, 1995-2015 (THOUSANDS) 26
FIGURE 4 BIRTHS FOR SELECTED COUNTRIES, 1995-2015 (THOUSANDS) 26
Chapter-4: ISSUES AFFECTING THE MARKET
GENETIC RESEARCH 27
GENE THERAPY COMPARED TO CELL THERAPY 27
ISSUES WITH HUMAN CLONING 28
LEGISLATIVE ISSUES 28
THE GENETIC INFORMATION NONDISCRIMINATION ACT (GINA) 28
GERMAN LEGISLATION 29
AUSTRALIAN GENETIC TESTING LAW 29
EUROPEAN UNION ACTION IN THE FIELD OF RARE DISEASES 30
GENETICS CLINICS 30
TABLE 7 NUMBER OF GENETIC CLINICS BY COUNTRY 31
GENOME SEQUENCING TECHNOLOGY 31
TECHNOLOGICAL APPLICATIONS OF GENOME SEQUENCING 32
SEQUENCING COSTS 32
Chapter-5: GENE MUTATION DISORDERS
DESCRIPTIONS OF GENE MUTATION DISORDERS 33
CYSTIC FIBROSIS 33
Treatment Options 34
Key Products 34
TABLE 8 PHARMACEUTICAL TREATMENTS FOR CYSTIC FIBROSIS 34
New Developments 34
New Developments (Continued) 35
TABLE 9 CYSTIC FIBROSIS DEVELOPMENTS BY PHASE OF DEVELOPMENT 36
TABLE 9 (CONTINUED) 37
FABRY DISEASE 37
Treatment Options 37
Key Products 38
TABLE 10 PHARMACEUTICAL TREATMENTS FOR FABRY DISEASE 38
New Developments 38
TABLE 11 FABRY DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT 38
GAUCHER'S DISEASE 39
Treatment Options 39
Key Products 40
TABLE 12 PHARMACEUTICAL TREATMENTS FOR GAUCHER'S DISEASE 40
New Developments 40
TABLE 13 GAUCHER'S DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT 41
HEMOPHILIA AND OTHER INHERITED BLEEDING DISORDERS 41
Treatment Options 41
Key Products 41
Key Products (Continued) 42
TABLE 14 BIOLOGICAL AND PHARMACEUTICAL TREATMENTS FOR HEMOPHILIA AND OTHERS 43
TABLE 15 EXPANDED INDICATIONS AND PRODUCT CHANGES 2005-2009 44
New Developments 45
TABLE 16 HEMOPHILIA AND RELATED BLOOD DISORDER DEVELOPMENTS BY PHASE OF DEVELOPMENT 46
HUNTINGTON'S DISEASE 46
Treatment Options 47
Key Products 47
TABLE 17 PHARMACEUTICAL TREATMENTS FOR HUNTINGTON'S DISEASE 47
New Developments 48
TABLE 18 HUNTINGTON'S DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT 48
HUNTER SYNDROME 48
Treatment Options 49
Key Products 49
TABLE 19 PHARMACEUTICAL TREATMENTS FOR HUNTER SYNDROME 49
New Developments 49
TABLE 20 HUNTER SYNDROME DEVELOPMENTS BY PHASE OF DEVELOPMENT 50
HURLER SYNDROME 50
Treatment Options 50
Key Products 51
TABLE 21 PHARMACEUTICAL TREATMENTS FOR HURLER SYNDROME 51
New Developments 51
MAROTEAUX-LAMY SYNDROME 51
Treatment Options 51
Key Products 52
TABLE 22 PHARMACEUTICAL TREATMENTS FOR MAROTEAUX-LAMY SYNDROME 52
PHENYLKETONURIA (PKU) 52
Treatment Options 53
Key Products 53
TABLE 23 PHARMACEUTICAL TREATMENTS FOR PKU 53
New Developments 54
TABLE 24 PKU DEVELOPMENTS BY PHASE OF DEVELOPMENT 54
POMPE DISEASE 54
Treatment Options 54
Key Products 55
TABLE 25 PHARMACEUTICAL TREATMENTS FOR POMPE DISEASE 55
New Developments 55
TABLE 26 POMPE DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT 55
SICKLE CELL DISEASE 55
Treatment Options 56
Key Products 56
TABLE 27 PHARMACEUTICAL TREATMENTS FOR SICKLE CELL DISEASE 57
New Developments 57
TABLE 28 SICKLE CELL DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT 57
TAY-SACHS DISEASE 57
Treatment Options 58
Key Products 58
New Developments 58
TABLE 29 TAY-SACHS DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT 59
WILSON'S DISEASE 59
Treatment Options 59
Key Products 59
TABLE 30 PHARMACEUTICAL TREATMENTS FOR WILSON'S DISEASE 60
New Developments 60
TABLE 31 WILSON'S DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT 61
OTHER APPROVED PRODUCTS FOR RARE GENETIC DISORDERS 61
OTHER NEW DEVELOPMENT PROJECTS 61
PHASE II 61
PHASE I 62
PRECLINICAL 62
MUSCULAR DYSTROPHY FOCUS: PRECLINICAL THROUGH PHASE III 63
AVI-4658 63
AVI-5038 64
Catena/Sovrima 64
PRO044 64
PTC-124 64
SNT-317/omigapil 64
STATISTICS 65
CYSTIC FIBROSIS 65
TABLE 32 CYSTIC FIBROSIS STATISTICS 65
GAUCHER'S DISEASE 65
TABLE 33 GAUCHER'S DISEASE STATISTICS 66
PHENYLKETONURIA 66
TAY-SACHS DISEASE 66
TABLE 34 TAY-SACHS DISEASE STATISTICS 67
SICKLE CELL DISEASE 67
TABLE 35 SICKLE-CELL DISEASE STATISTICS 67
HEMOPHILIA 67
HUNTINGTON'S DISEASE 68
MARKET SIZE AND GROWTH 68
TABLE 36 GLOBAL GENE MUTATION PRODUCT REVENUES BY DISORDER THROUGH 2014 ($ MILLIONS) 69
FIGURE 5 GLOBAL GENE MUTATION PRODUCT REVENUES BY DISORDER, 2007-2014 ($ MILLIONS) 70
COMPETITIVE STRUCTURE AND MARKET SHARE 70
CYSTIC FIBROSIS MARKET 70
TABLE 37 GLOBAL PRODUCT REVENUES FOR CYSTIC FIBROSIS TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS) 71
FIGURE 6 GLOBAL PRODUCT REVENUES FOR CYSTIC FIBROSIS TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 72
FABRY DISEASE MARKET 72
TABLE 38 GLOBAL PRODUCT REVENUES FOR FABRY DISEASE TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS) 73
FIGURE 7 GLOBAL PRODUCT REVENUES FOR FABRY DISEASE TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 73
GAUCHER'S DISEASE MARKET 74
TABLE 39 GLOBAL PRODUCT REVENUES FOR GAUCHER'S DISEASE TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS) 74
FIGURE 8 GLOBAL PRODUCT REVENUES FOR GAUCHER'S DISEASE TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 75
HEMOPHILIA AND RELATED BLOOD DISORDER MARKET 75
Hemophilia and Related Blood ... (Continued) 76
TABLE 40 GLOBAL PRODUCT REVENUES FOR HEMOPHILIA TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS) 77
FIGURE 9 GLOBAL PRODUCT REVENUES FOR HEMOPHILIA TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 77
HUNTINGTON'S DISEASE MARKET 78
TABLE 41 GLOBAL PRODUCT REVENUES FOR HUNTINGTON'S DISEASE TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS) 78
FIGURE 10 GLOBAL PRODUCT REVENUES FOR HUNTINGTON'S DISEASE TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 79
HUNTER SYNDROME MARKET 79
TABLE 42 GLOBAL PRODUCT REVENUES FOR HUNTER SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS) 80
FIGURE 11 GLOBAL PRODUCT REVENUES FOR HUNTER SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 80
HURLER SYNDROME MARKET 80
TABLE 43 GLOBAL PRODUCT REVENUES FOR HURLER SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS) 81
FIGURE 12 GLOBAL PRODUCT REVENUES FOR HURLER SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 81
MAROTEAUX-LAMY SYNDROME MARKET 81
TABLE 44 GLOBAL PRODUCT REVENUES FOR MAROTEAUX-LAMY SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS) 82
FIGURE 13 GLOBAL PRODUCT REVENUES FOR MAROTEAUX-LAMY SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 82
PHENYLKETONURIA (PKU) MARKET 83
TABLE 45 GLOBAL PRODUCT REVENUES FOR PKU TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS) 83
FIGURE 14 GLOBAL PRODUCT REVENUES FOR PKU TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 83
POMPE DISEASE MARKET 84
TABLE 46 GLOBAL PRODUCT REVENUES FOR POMPE DISEASE TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS) 84
FIGURE 15 GLOBAL PRODUCT REVENUES FOR POMPE DISEASE TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 84
Chapter-6: CHROMOSONAL DISORDERS
DESCRIPTIONS OF CHROMOSONAL DISORDERS 85
DOWN SYNDROME 85
Treatment Options 85
New Developments 86
TABLE 47 DOWN SYNDROME DEVELOPMENTS BY PHASE OF DEVELOPMENT 86
KLINEFELTER SYNDROME 86
Treatment Options 87
Key Products 87
TABLE 48 EXAMPLES OF PHARMACEUTICAL TREATMENTS FOR KLINEFELTER SYNDROME 87
New Developments 88
TABLE 49 EXAMPLES OF TESTOSTERONE REPLACEMENT DEVELOPMENTS BY PHASE OF DEVELOPMENT 88
PRADER-WILLI SYNDROME 88
Treatment Options 88
Key Products 89
TABLE 50 PHARMACEUTICAL TREATMENTS FOR PRADER-WILLI SYNDROME 89
New Developments 89
TURNER SYNDROME 90
Treatment Options 90
Key Products 91
TABLE 51 FDA APPROVED BIOPHARMACEUTICAL TREATMENTS FOR TURNER'S SYNDROME 92
TABLE 51 (CONTINUED) 93
New Developments 93
TABLE 52 TURNER SYNDROME DEVELOPMENTS BY PHASE OF DEVELOPMENT 93
STATISTICS 93
DOWN SYNDROME 93
TABLE 53 DOWN SYNDROME RISK AS A COMPARISON TO MATERNAL AGE 94
KLINEFELTER SYNDROME 94
PRADER-WILLI SYNDROME 94
TURNER SYNDROME 94
MARKET SIZE AND GROWTH 94
TABLE 54 GLOBAL CHROMOSONAL DISORDER PRODUCT REVENUES* BY DISORDER, THROUGH 2014 ($ MILLIONS) 95
FIGURE 16 GLOBAL CHROMOSONAL DISORDER PRODUCT REVENUES BY DISORDER, 2007-2014 ($ MILLIONS) 95
COMPETITIVE STRUCTURE AND MARKET SHARE 96
KLINEFELTER SYNDROME 96
TABLE 55 GLOBAL PRODUCT REVENUES FOR KLINEFELTER SYNDROME TREATMENTS, THROUGH 2014 ($ MILLIONS) 96
FIGURE 17 GLOBAL PRODUCT REVENUES FOR KLINEFELTER SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 97
PRADER-WILLI SYNDROME 97
TABLE 56 GLOBAL PRODUCT REVENUES* FOR PRADER-WILLI SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS) 98
FIGURE 18 GLOBAL PRODUCT REVENUES FOR PRADER-WILLI SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 98
TURNER'S SYNDROME 98
TABLE 57 GLOBAL PRODUCT REVENUES* FOR TURNER'S SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS) 99
FIGURE 19 GLOBAL PRODUCT REVENUES FOR TURNER'S SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS) 100
Chapter-7: MARKET SUMMARY
TOTAL GLOBAL MARKET FOR GENETIC DISORDERS 101
TABLE 58 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY MAJOR MARKET SEGMENT, THROUGH 2014 ($ MILLIONS) 101
FIGURE 20 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY MAJOR MARKET SEGMENT, 2007-2014 ($ MILLIONS) 102
TOTAL GLOBAL MARKET AND PERCENT DISTRIBUTION BY SELECTED GENETIC DISORDER 102
TABLE 59 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY DISORDER THROUGH 2014 ($ MILLIONS) 103
FIGURE 21 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY DISORDER, 2007-2014 ($ MILLIONS) 104
GEOGRAPHICAL ANALYSIS OF GENETIC DISORDER TREATMENTS 104
TABLE 60 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY SELECTED COUNTRY, THROUGH 2014 ($ MILLIONS) 105
FIGURE 22 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY SELECTED COUNTRY, 2007-2014 ($ MILLIONS) 106
MAJOR INDUSTRY PARTICIPANTS BY PRODUCT SEGMENT 106
MAJOR INDUSTRY PARTICIPANTS ...(CONTINUED) 107
TABLE 61 MAJOR INDUSTRY PARTICIPANTS BY PRODUCT SEGMENTS AND SALES GROWTH, THROUGH 2014 ($ MILLIONS) 108
TABLE 61 (CONTINUED) 109
Chapter-8: TECHNOLOGY
PATENT ANALYSIS 110
PATENTS AND GENETICS 110
PATENT EVALUATION BY GENETIC CONDITION 111
TABLE 62 PATENT INFORMATION BY GENETIC CONDITION 112
TABLE 62 (CONTINUED) 113
TABLE 62 (CONTINUED) 114
TABLE 62 (CONTINUED) 115
TABLE 62 (CONTINUED) 116
FIGURE 23 PERCENT OF PATENTS BY GENETIC CONDITION (%) 116
FIGURE 23 (CONTINUED) 117
PATENT EVALUATION BY ASSIGNEE 117
TABLE 63 GENETIC PATENT ASSIGNEES 117
TABLE 63 (CONTINUED) 118
TABLE 63 (CONTINUED) 119
Bristol-Myers Squibb 120
Eli Lilly 120
G. D. Searle & Co./Monsanto Company 120
Genzyme Corp. 120
Purdue Pharma 120
Sigma-Tau Industrie Farmaceutiche Riunite S.p.A. 121
Transkaryotic Therapies 121
Wyeth 121
Chapter-9: COMPANY PROFILES
BAXTER INTERNATIONAL, INC. 122
TABLE 64 BAXTER'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 123
FIGURE 24 BAXTER'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 124
BAYER AG 124
TABLE 65 BAYER'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 125
FIGURE 25 BAYER'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 126
BIOMARIN PHARMACEUTICAL, INC. 126
TABLE 66 BIOMARIN'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 127
FIGURE 26 BIOMARIN'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 127
CANGENE CORP. 128
TABLE 67 CANGENE'S COMPANY REVENUES, 2007-2009* ($ MILLIONS) 129
FIGURE 27 CANGENE'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 129
CSL BEHRING (CSL LIMITED) 129
TABLE 68 CSL'S COMPANY REVENUES, 2007-2009* ($ MILLIONS) 130
FIGURE 28 CSL'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 130
ELI LILLY 131
TABLE 69 ELI LILLY'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 131
FIGURE 29 ELI LILLY'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 132
ENDO PHARMACEUTICALS 132
TABLE 70 ENDO'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 133
FIGURE 30 ENDO'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 134
GENENTECH INC. 134
TABLE 71 GENENTECH AND ROCHE COMPANY REVENUES, 2007-2009 ($ MILLIONS) 135
FIGURE 31 GENENTECH AND ROCHE COMPANY REVENUES, 2007-2009 ($ MILLIONS) 135
GENZYME 135
TABLE 72 GENZYME'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 136
FIGURE 32 GENZYME'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 137
H. LUNDBECK A/S 137
TABLE 73 LUNDBECK'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 138
FIGURE 33 LUNDBECK'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 138
NOVARTIS INTERNATIONAL AG/SANDOZ 138
NOVARTIS INTERNATIONAL AG/... (CONTINUED) 139
TABLE 74 NOVARTIS' COMPANY REVENUES, 2007-2009 ($ MILLIONS) 140
FIGURE 34 NOVARTIS' COMPANY REVENUES, 2007-2009 ($ MILLIONS) 140
NOVO NORDISK A/S 140
TABLE 75 NOVO NORDISK'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 141
FIGURE 35 NOVO NORDISK'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 141
PFIZER, INC/WYETH 142
TABLE 76 PFIZER'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 143
FIGURE 36 PFIZER'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 143
SHIRE PLC 143
TABLE 77 SHIRE'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 144
FIGURE 37 SHIRE'S COMPANY REVENUES, 2007-2009 ($ MILLIONS) 144
Chapter-10: APPENDIX
APPENDIX I: COMPANIES WITH GENETIC RESEARCH PROJECTS 145
ADEONA PHARMACEUTICALS, INC. 145
AEROVANCE, INC. 145
ALDAGEN, INC. 145
AMICUS THERAPEUTICS, INC. 145
AOP ORPHAN PHARMACEUTICALS AG 145
APPLIED GENETIC TECHNOLOGIES CORP. 145
AVI BIOPHARMA 145
AVICENA GROUP, INC. 146
BAYER SCHERING PHARMA AG 146
BIOGEN IDEC 146
BIOMARIN PHARMACEUTICAL INC. 146
COPERNICUS THERAPEUTICS, INC 146
CSL BEHRING 146
EISAI, INC. 146
GILEAD SCIENCES, INC. 147
GLAXOSMITHKLINE PLC 147
HOLLIS-EDEN PHARMACEUTICALS, INC. 147
INSPIRE PHARMACEUTICALS, INC. 147
IPSEN 147
KALOBIOS PHARMACEUTICALS, INC. 147
LANTIBIO, INC. 147
MPEX PHARMACEUTICALS, INC. 148
NEUROSEARCH A/S 148
NORTHFIELD LABORATORIES, INC. 148
NOVO NORDISK A/S 148
PFIZER, INC. 148
PHARMAXIS LTD 148
PROSENSA HOLDING B.V. 149
PROTALIX BIOTHERAPEUTICS 149
PTC THERAPEUTICS, INC. 149
QUATRX PHARMACEUTICALS 149
REGENERX BIOPHARMACEUTICALS, INC. 149
REPROS THERAPEUTICS INC. 149
SANTHERA PHARMACEUTICALS HOLDING AG 150
SHIRE PLC 150
SUCAMPO PHARMACEUTICALS, INC. 150
TRANSAVE, INC. 150
VERTEX PHARMACEUTICALS, INC. 150
APPENDIX II: ASSOCIATIONS AND ORGANIZATIONS 151
ANGELMAN SYNDROME FOUNDATION, INC. 151
CANADIAN SOCIETY FOR MUCOPOLYSACCHARIDE AND RELATED DISEASES, INC. 151
CHILDREN LIVING WITH INHERITED METABOLIC DISEASES (CLIMB) 152
CELIAC DISEASE FOUNDATION 152
CELIAC SPRUE ASSOCIATION 152
CURE CMD (CONGENITAL MUSCULAR DYSTROPHY) 152
CYSTIC FIBROSIS FOUNDATION 152
CYSTIC FIBROSIS RESEARCH, INC. 152
CYSTIC FIBROSIS TRUST 153
CYSTIC FIBROSIS WORLDWIDE 153
DOWN'S SYNDROME ASSOCIATION (UK) 153
FABRY SUPPORT & INFORMATION GROUP 153
GAUCHERS ASSOCIATION (UK) 153
HIDE & SEEK FOUNDATION FOR LYSOSOMAL DISEASE RESEARCH 153
HEMOPHILIA FEDERATION OF AMERICA 154
HUNTINGTON'S ASSOCIATION OF SOUTH AFRICA POSTBOX 4640 154
HUNTINGTON'S DISEASE SOCIETY OF AMERICA 154
HUNTINGTON SOCIETY OF CANADA 154
INTERNATIONAL CENTER FOR FABRY DISEASE 154
INSTITUTO DE ERRORES INNATOS DEL METABOLISMO 155
KLINEFELTER SYNDROME & ASSOCIATES (KS&A) 155
MARCH OF DIMES BIRTH DEFECTS FOUNDATION 155
MUSCULAR DYSTROPHY ASSOCIATION 155
MUSCULAR DYSTROPHY LIMITED (AUSTRALIA) 155
MUSCULAR DYSTROPHY ASSOCIATION OF NEW ZEALAND, INC. 155
MUSCULAR DYSTROPHY CAMPAIGN 156
MUSCULAR DYSTROPHY CANADA 156
MUSCULAR DYSTROPHY IRELAND 156
NATIONAL FOUNDATION FOR CELIAC AWARENESS (NFCA) 156
NATIONAL GAUCHER'S FOUNDATION 156
NATIONAL HEMOPHILIA FOUNDATION 156
NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE (NINDS) 157
NATIONAL MPS SOCIETY (HUNTER SYNDROME) 157
NATIONAL MPS (MUCOPOLYSACCHARIDOSES/ MUCOLIPIDOSES) SOCIETY, INC. 157
NATIONAL ORGANIZATION FOR RARE DISORDERS (NORD) 157
NATIONAL TAY-SACHS AND ALLIED DISEASES ASSOCIATION, INC. 157
PRADER-WILLI SYNDROME ASSOCIATION (UK) 158
THE PRADER-WILLI SYNDROME ASSOCIATION (USA) 158
SICKLE CELL ASSOCIATION 158
SICKLE CELL ASSOCIATION OF ONTARIO 158
SICKLE CELL ASSOCIATION OF TEXAS GULF COAST 158
SICKLE CELL DISEASE ASSOCIATION OF PIEDMONT 158
SICKLE CELL DISEASE FOUNDATION OF CALIFORNIA 158
SOCIETY FOR MUSCULAR DYSTROPHY INFORMATION INTERNATIONAL 159
TURNER'S SYNDROME SOCIETY 159
TURNER SYNDROME SUPPORT SOCIETY (UK) 159
U.S. NATIONAL LIBRARY OF MEDICINE 159
APPENDIX III: INTERNATIONAL LOCATIONS OF GENETIC CLINICS 160
ARGENTINA 160
AUSTRALIA 160
AUSTRIA 161
BAHRAIN 161
BELGIUM 161
BRAZIL 162
CANADA 162
CHILE 163
CZECH REPUBLIC 163
DENMARK 163
FRANCE 163
GERMANY 164
GREECE 164
INDIA 164
ISRAEL 164
ITALY 165
JAPAN 165
NETHERLANDS 165
NEW ZEALAND 166
PORTUGAL 166
RUSSIA 166
SAUDI ARABIA 166
SOUTH KOREA 166
SPAIN 167
TURKEY 167
UNITED KINGDOM 167
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Biotechnology Industry: New Developments in Treating Genetic Disorders: The Global Market
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