The global duchenne muscular dystrophy treatment market is expected to reach USD 8,654,688.51 thousand by 2030 from USD 2,595,090.00 thousand in 2022, growing at a CAGR of 16.8% in the forecast period of 2023 to 2030.
Market Segmentation
Global Duchenne Muscular Dystrophy Treatment Market, By Treatment Type (Molecular-Based Therapies, Steroid Therapy, and Others), Therapy (Exon Skipping Approach, Mutation Suppression, and Dystrophin-Targeted Therapies), Route of Administration (Oral, Parenteral, and Others), End User (Hospitals, Specialty Clinics, Homecare, and Others), Distribution Channel (Hospital Pharmacy, Retail Pharmacy, and Online Pharmacy), Country (U.S., Canada, Mexico, Germany, France, U.K., Italy, Spain, Russia, Turkey, Belgium, Netherlands, Switzerland, Rest of Europe, Japan, China, South Korea, India, Australia, Singapore, Thailand, Malaysia, Indonesia, Philippines, Rest of Asia-Pacific, Brazil, Argentina, Rest of South America, South Africa, Saudi Arabia, U.A.E., Egypt, Israel, and Rest of Middle East and Africa) - Industry Trends and Forecast to 2030.
Overview of Global Duchenne Muscular Dystrophy Treatment Market Dynamics
- Driver
- Increasing prevalence and impact of Duchenne Muscular Dystrophy (DMD) disease
- Restraint
- High treatment cost of DMD disorder
- Opportunity
- Strategic initiatives by key market players
Market Players
Some of the key market players for global duchenne muscular dystrophy treatment market are:
- Sarepta Therapeutics, Inc.
- Solid Biosciences Inc.
- Capricor Therapeutics, Inc.
- Dyne Therapeutics
- BioMarin
- Stealth BioTherapeutics Inc
- Avidity Biosciences
- ReveraGen
- BioPharma, Inc.
- TAIHO PHARMACEUTICAL CO., LTD.
- Akashi RX
- ITALFARMACO S.p.
- NS Pharma, Inc.
- PTC Therapeutics.
- Pfizer Inc.
- FibroGen, Inc.
- SANTHERA PHARMACEUTICALS
- F. Hoffmann-La Roche Ltd
TABLE OF CONTENTS
1 INTRODUCTION 45
- 1.1 OBJECTIVES OF THE STUDY 45
- 1.2 MARKET DEFINITION 45
- 1.3 OVERVIEW OF THE GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET 45
- 1.4 CURRENCY AND PRICING 47
- 1.5 LIMITATIONS 47
- 1.6 MARKETS COVERED 47
2 MARKET SEGMENTATION 49
- 2.1 MARKETS COVERED 49
- 2.2 GEOGRAPHICAL SCOPE 50
- 2.3 YEARS CONSIDERED FOR THE STUDY 51
- 2.4 DBMR TRIPOD DATA VALIDATION MODEL 52
- 2.5 PRIMARY INTERVIEWS WITH KEY OPINION LEADERS 55
- 2.6 MULTIVARIATE MODELLING 56
- 2.7 TREATMENT TYPE SEGMENT LIFELINE CURVE 56
- 2.8 MARKET END USER COVERAGE GRID 57
- 2.9 DBMR MARKET POSITION GRID 58
- 2.10 VENDOR SHARE ANALYSIS 59
- 2.11 SECONDARY SOURCES 60
- 2.12 ASSUMPTIONS 60
3 EXECUTIVE SUMMARY 61
4 PREMIUM INSIGHTS 63
- 4.1 PESTEL'S MODEL 64
- 4.2 PORTER'S FIVE FORCES MODEL 65
- 4.3 PRICING ANALYSIS 65
5 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET: REGULATIONS 67
- 5.1 REGULATIONS IN U.S. 67
- 5.2 REGULATTIONS IN EUROPE 68
- 5.3 REGULATTIONS IN AUSTRALIA 68
- 5.4 REGULATIONS IN SOUTH AFRICA 68
- 5.5 REGULATIONS IN BRAZIL 69
6 MARKET OVERVIEW 70
- 6.1 DRIVERS 72
- 6.1.1 INCREASING PREVALENCE AND IMPACT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) DISEASE 72
- 6.1.2 INTRODUCTION OF NOVEL THERAPIES FOR DMD DISORDER 72
- 6.1.3 RISING AWARENESS FOR DMD TREATMENTS 73
- 6.1.4 INCREASE IN THE NUMBER OF CLINICAL TRIALS IS A RECENT TREND 74
- 6.2 RESTRAINTS 75
- 6.2.1 LIMITED AVAILABILITY OF DMD TREATMENTS DUE TO LACK OF ADVANCE TECHNOLOGY 75
- 6.2.2 HIGH TREATMENT COST OF DMD DISORDER 76
- 6.3 OPPORTUNITIES 77
- 6.3.1 DEVELOPMENT OF HEALTHCARE INFRASTRUCTURE AND INCREASING INVESTMENT 77
- 6.3.2 RISING PATIENT INCLINATION TOWARDS PERSONALIZED AND EFFECTIVE THERAPIES 77
- 6.3.3 STRATEGIC INITIATIVES BY THE KEY MARKET PLAYERS 78
- 6.3.4 SUPPORT FROM PATIENT ADVOCACY GROUPS ON THE REGULATORY APPROVAL PROCESS 78
- 6.4 CHALLENGES 79
- 6.4.1 LACK OF STANDARDIZATION IN DMD DIAGNOSIS 79
- 6.4.2 ETHICAL CONSIDERATIONS RELATED TO PERMANENT ALTERATION OF A PATIENT'S GENETIC CODE 80
7 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, BY TREATMENT TYPE 81
- 7.1 OVERVIEW 82
- 7.2 MOLECULAR-BASED THERAPIES 85
- 7.2.1 ANTISENSE OLIGONUCLEOTIDE THERAPY 86
- 7.2.1.1 EXONDYS 51 86
- 7.2.1.2 AMONDYS 45 87
- 7.2.1.3 VYONDYS 53 87
- 7.3 NONSENSE MUTATION 87
- 7.4 STEROID THERAPY 88
- 7.4.1 PREDNISONE 88
- 7.4.2 DEFLAZACORT 88
- 7.5 OTHERS 89
8 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, BY THERAPY 90
- 8.1 OVERVIEW 91
- 8.2 EXON SKIPPING APPROACH 94
- 8.2.1 MULTI-EXON SKIPPING APPROACH 94
- 8.2.2 SINGLE-EXON SKIPPING APPROACH 95
- 8.3 MUTATION SUPPRESSION 95
- 8.4 DYSTROPHIN-TARGETED THERAPIES 96
- 8.4.1 GENE THERAPIES 96
- 8.4.2 CELL THERAPIES 97
- 8.4.2.1 Gene Editing 97
- 8.4.2.2 Gene Addition 97
9 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, BY ROUTE OF ADMINISTRATION 98
- 9.1 OVERVIEW 99
- 9.2 PARENTERAL 102
- 9.3 ORAL 103
- 9.4 OTHERS 104
10 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, BY END USER 105
- 10.1 OVERVIEW 106
- 10.2 HOSPITALS 109
- 10.3 SPECIALTY CLINICS 110
- 10.4 HOMECARE 111
- 10.5 OTHERS 112
11 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, BY DISTRIBUTION CHANNEL 113
- 11.1 OVERVIEW 114
- 11.2 HOSPITAL PHARMACY 117
- 11.3 RETAIL PHARMACY 118
- 11.4 ONLINE PHARMACY 119
12 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, BY REGION 120
- 12.1 OVERVIEW 121
- 12.2 NORTH AMERICA 124
- 12.2.1 U.S. 129
- 12.2.2 CANADA 134
- 12.2.3 MEXICO 139
- 12.3 EUROPE 145
- 12.3.1 GERMANY 151
- 12.3.2 FRANCE 156
- 12.3.3 U.K. 161
- 12.3.4 ITALY 166
- 12.3.5 SPAIN 171
- 12.3.6 RUSSIA 176
- 12.3.7 TURKEY 181
- 12.3.8 BELGIUM 186
- 12.3.9 NETHERLANDS 191
- 12.3.10 SWITZERLAND 196
- 12.3.11 REST OF EUROPE 201
- 12.4 ASIA-PACIFIC 202
- 12.4.1 CHINA 207
- 12.4.2 JAPAN 212
- 12.4.3 INDIA 217
- 12.4.4 SOUTH KOREA 222
- 12.4.5 AUSTRALIA 227
- 12.4.6 SINGAPORE 232
- 12.4.7 THAILAND 237
- 12.4.8 MALAYSIA 242
- 12.4.9 INDONESIA 247
- 12.4.10 PHILIPPINES 252
- 12.4.11 REST OF ASIA-PACIFIC 257
- 12.5 SOUTH AMERICA 258
- 12.5.1 BRAZIL 263
- 12.5.2 ARGENTINA 268
- 12.5.3 REST OF SOUTH AMERICA 273
- 12.6 MIDDLE EAST AND AFRICA 274
- 12.6.1 SOUTH AFRICA 279
- 12.6.2 SAUDI ARABIA 284
- 12.6.3 U.A.E 289
- 12.6.4 EGYPT 294
- 12.6.5 ISRAEL 299
- 12.6.6 REST OF MIDDLE EAST AND AFRICA 304
13 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, COMPANY LANDSCAPE 305
- 13.1 COMPANY SHARE ANALYSIS: GLOBAL 305
14 NORTH AMERICA DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, COMPANY LANDSCAPE 306
- 14.1 COMPANY SHARE ANALYSIS: NORTH AMERICA 306
15 EUROPE DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, COMPANY LANDSCAPE 307
- 15.1 COMPANY SHARE ANALYSIS: EUROPE 307
16 ASIA-PACIFIC DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, COMPANY LANDSCAPE 308
- 16.1 COMPANY SHARE ANALYSIS: ASIA-PACIFIC 308
17 SWOT ANALYSIS 309
18 COMPANY PROFILES 310
- 18.1 SAREPTA THERAPEUTICS, INC. 310
- 18.1.1 COMPANY SNAPSHOT 310
- 18.1.1 REVENUE ANALYSIS 311
- 18.1.2 COMPANY SHARE ANALYSIS 311
- 18.1.3 PRODUCT PORTFOLIO 312
- 18.1.4 PIPELINE PORTFOLIO 312
- 18.1.5 RECENT DEVELOPMENTS 312
- 18.2 F. HOFFMANN-LA ROCHE LTD 314
- 18.2.1 COMPANY SNAPSHOT 314
- 18.2.2 REVENUE ANALYSIS 314
- 18.2.3 COMPANY SHARE ANALYSIS 315
- 18.2.4 PRODUCT PORTFOLIO 315
- 18.2.5 PIPELINE PORTFOLIO 315
- 18.2.6 RECENT DEVELOPMENTS 315
- 18.3 PTC THERAPEUTICS. 316
- 18.3.1 COMPANY SNAPSHOT 316
- 18.3.2 REVENUE ANALYSIS 316
- 18.3.3 COMPANY SHARE ANALYSIS 317
- 18.3.4 PRODUCT PORTFOLIO 317
- 18.3.5 RECENT DEVELOPMENT 317
- 18.4 PFIZER INC. 318
- 18.4.1 COMPANY SNAPSHOT 318
- 18.4.2 REVENUE ANALYSIS 318
- 18.4.3 COMPANY SHARE ANALYSIS 319
- 18.4.4 PIPELINE PORTFOLIO 319
- 18.4.5 PRODUCT PORTFOLIO 319
- 18.4.6 RECENT DEVELOPMENT 319
- 18.5 AKASHI RX 320
- 18.5.1 COMPANY SNAPSHOT 320
- 18.5.2 PIPELINE PORTFOLIO 320
- 18.5.3 RECENT DEVELOPMENTS 320
- 18.6 AVIDITY BIOSCIENCES 321
- 18.6.1 COMPANY SNAPSHOT 321
- 18.6.2 REVENUE ANALYSIS 321
- 18.6.3 PIPELINE PORTFOLIO 322
- 18.6.4 RECENT DEVELOPMENTS 322
- 18.7 BIOMARIN 323
- 18.7.1 COMPANY SNAPSHOT 323
- 18.7.2 REVENUE ANALYSIS 323
- 18.7.3 PIPELINE PORTFOLIO 324
- 18.7.4 RECENT DEVELOPMENTS 324
- 18.8 CAPRICOR THERAPEUTICS, INC. 325
- 18.8.1 COMPANY SNAPSHOT 325
- 18.8.2 PIPELINE PORTFOLIO 325
- 18.8.3 RECENT DEVELOPMENTS 326
- 18.9 DYNE THERAPEUTICS 327
- 18.9.1 COMPANY SNAPSHOT 327
- 18.9.2 PIPELINE PORTFOLIO 327
- 18.9.3 RECENT DEVELOPMENTS 328
- 18.10 FIBROGEN, INC. 329
- 18.10.1 COMPANY SNAPSHOT 329
- 18.10.2 REVENUE ANALYSIS 329
- 18.10.3 PIPELINE PORTFOLIO 330
- 18.10.4 RECENT DEVELOPMENT 330
- 18.11 ITALFARMACO S.P.A. 331
- 18.11.1 COMPANY SNAPSHOT 331
- 18.11.2 PIPELINE PORTFOLIO 331
- 18.11.3 RECENT DEVELOPMENT 331
- 18.12 NS PHARMA, INC. 332
- 18.12.1 COMPANY SNAPSHOT 332
- 18.12.2 PIPELINE PORTFOLIO 332
- 18.12.3 RECENT DEVELOPMENTS 333
- 18.13 REVERAGEN BIOPHARMA, INC. 334
- 18.13.1 COMPANY SNAPSHOT 334
- 18.13.2 PIPELINE PORTFOLIO 334
- 18.13.3 RECENT DEVELOPMENT 334
- 18.14 SANTHERA PHARMACEUTICALS 335
- 18.14.1 COMPANY SNAPSHOT 335
- 18.14.2 REVENUE ANALYSIS 335
- 18.14.3 PIPELINE PORTFOLIO 336
- 18.14.4 RECENT DEVELOPMENT 336
- 18.15 TAIHO PHARMACEUTICAL CO., LTD. 337
- 18.15.1 COMPANY SNAPSHOT 337
- 18.15.2 PIPELINE PORTFOLIO 337
- 18.15.3 RECENT DEVELOPMENTS 337
- 18.16 SOLID BIOSCIENCES INC. 338
- 18.16.1 COMPANY SNAPSHOT 338
- 18.16.2 PIPELINE PORTFOLIO 338
- 18.16.3 RECENT DEVELOPMENT 338
- 18.17 STEALTH BIOTHERAPEUTICS INC 339
- 18.17.1 COMPANY SNAPSHOT 339
- 18.17.2 PIPELINE PORTFOLIO 339
- 18.17.3 RECENT DEVELOPMENT 339
19 QUESTIONNAIRE 340
20 RELATED REPORTS 343