Table of Contents 2
List of Tables 3
List of Figures 4
Report Guidance 5
GlobalData Clinical Trials Report Coverage 6
Clinical Trials by Region 7
Clinical Trials and Average Enrollment by Country 8
Top Five Countries Contributing to Clinical Trials in Asia-Pacific 11
Top Five Countries Contributing to Clinical Trials in Europe 12
Top Countries Contributing to Clinical Trials in North America 13
Top Countries Contributing to Clinical Trials in Middle East and Africa 14
Top Countries Contributing to Clinical Trials in Central and South America 15
Clinical Trials by G7 Countries: Proportion of Spinal Muscular Atrophy (SMA) to Central Nervous System Clinical Trials 16
Clinical Trials by Phase in G7 Countries 17
Clinical Trials in G7 Countries by Trial Status 18
Clinical Trials by E7 Countries: Proportion of Spinal Muscular Atrophy (SMA) to Central Nervous System Clinical Trials 19
Clinical Trials by Phase in E7 Countries 20
Clinical Trials in E7 Countries by Trial Status 21
Clinical Trials by Phase 22
In Progress Trials by Phase 23
Clinical Trials by Trial Status 24
Clinical Trials by End Point Status 25
Subjects Recruited Over a Period of Time 26
Clinical Trials by Sponsor Type 27
Prominent Sponsors 28
Top Companies Participating in Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials 30
Prominent Drugs 32
Latest Clinical Trials News on Spinal Muscular Atrophy (SMA) 33
Jun 16, 2018: Updated Preliminary Data from SMA FIREFISH Program in Type 1 Babies Presented at the CureSMA Conference 33
Jun 16, 2018: Cytokinetics Announces Data From Phase 2 Clinical Study of Reldesemtiv in Patients With Spinal Muscular Atrophy Presented at the 2018 Annual Cure SMA Conference 33
Jun 14, 2018: Exicure to Present Data at the Cure Spinal Muscular Atrophy Annual Conference in Dallas 34
Jun 13, 2018: Biogen Enhances Commitment to SMA Research with Data Presented at Annual SMA Conference 35
Jun 08, 2018: Cytokinetics Announces Data From Phase 2 Clinical Study of Reldesemtiv in Patients With Spinal Muscular Atrophy to be Presented at the 2018 Annual Cure SMA Conference on June 16, 2018 35
May 31, 2018: Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015 35
May 22, 2018: Biogen Releases Community Statement on Spinraza Access and New Data 36
Clinical Trial Profile Snapshots 37
Appendix 92
Abbreviations 92
Definitions 92
Research Methodology 93
Secondary Research 93
About GlobalData 94
Contact Us 94
Disclaimer 94
Source 94

List of Tables
Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials by Region, 2018* 7
Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials and Average Enrollment by Top Countries, 2018* 9
Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials In Progress by Top Countries, 2018* 10
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Asia-Pacific, Top Five Countries, 2018* 11
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Europe, Top Five Countries, 2018* 12
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, North America, Top Countries, 2018* 13
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Middle East and Africa, Top Countries, 2018* 14
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Central and South America, Top Countries, 2018* 15
Proportion of Spinal Muscular Atrophy (SMA) to Central Nervous System Clinical Trials, G7 Countries (%), 2018* 16
Spinal Muscular Atrophy (SMA) Therapeutics, G7 Countries, Clinical Trials by Phase, 2018* 17
Spinal Muscular Atrophy (SMA) Therapeutics, G7 Countries, Clinical Trials by Trial Status, 2018* 18
Proportion of Spinal Muscular Atrophy (SMA) to Central Nervous System Clinical Trials, E7 Countries (%), 2018* 19
Spinal Muscular Atrophy (SMA) Therapeutics, E7 Countries, Clinical Trials by Phase, 2018* 20
Spinal Muscular Atrophy (SMA) Therapeutics, E7 Countries, Clinical Trials by Trial Status, 2018* 21
Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials by Phase, 2018* 22
Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials In Progress by Phase 2018* 23
Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials by Trial Status, 2018* 24
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, by End Point Status, 2018* 25
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Average Enrollment Target Trends, 2013-2017 26
Spinal Muscular Atrophy (SMA) Therapeutics Market, Global, Clinical Trials by Sponsor Type, 2018* 27
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Key Sponsors, 2018* 29
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Top Companies by Phase, 2018* 31
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Ongoing Clinical Trials by Prominent Drugs, 2018* 32

List of Figures
Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials by Region (%), 2018* 7
Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials and Average Enrollment by Top Countries, 2018* 8
Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials In Progress by Top Countries, 2018* 10
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Asia-Pacific, Top Five Countries (%), 2018* 11
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Europe, Top Five Countries (%), 2018* 12
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, North America, Top Countries (%), 2018* 13
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Middle East and Africa, Top Countries (%), 2018* 14
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Central and South America, Top Countries (%), 2018* 15
Proportion of Spinal Muscular Atrophy (SMA) to Central Nervous System Clinical Trials, G7 Countries (%), 2018* 16
Spinal Muscular Atrophy (SMA) Therapeutics, G7 Countries, Clinical Trials by Phase, 2018* 17
Spinal Muscular Atrophy (SMA) Therapeutics, G7 Countries, Clinical Trials by Trial Status, 2018* 18
Proportion of Spinal Muscular Atrophy (SMA) to Central Nervous System Clinical Trials, E7 Countries (%), 2018* 19
Spinal Muscular Atrophy (SMA) Therapeutics, E7 Countries, Clinical Trials by Phase, 2018* 20
Spinal Muscular Atrophy (SMA) Therapeutics, E7 Countries, Clinical Trials by Trial Status, 2018* 21
Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials by Phase (%), 2018* 22
Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials In Progress by Phase, 2018* 23
Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials by Trial Status, 2018* 24
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, by End Point Status, 2018* 25
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Average Enrollment Target Trends, 2013-2017 26
Spinal Muscular Atrophy (SMA) Therapeutics Market, Global, Clinical Trials by Sponsor Type (%), 2018* 27
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Key Sponsors, 2018* 28
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Top Companies by Phase, 2018* 30
Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Ongoing Clinical Trials by Prominent Drugs, 2018* 32
GlobalData Methodology 93

Companies Mentioned
- F. Hoffmann-La Roche Ltd
- Biogen Inc
- AveXis Inc
- PTC Therapeutics Inc
- Ionis Pharmaceuticals Inc
- Cytokinetics Inc
- Cure SMA
- Astellas Pharma Inc
- Abbott Laboratories
- AlfaSigma SpA