Spinal Muscular Atrophy (SMA) Disease – Global Clinical Trials Review, H1 2020

GlobalData's clinical trial report, “Spinal Muscular Atrophy (SMA) Global Clinical Trials Review, H1, 2020" provides an overview of Spinal Muscular Atrophy (SMA) Clinical trials scenario. This report provides top line data relating to the clinical trials on Spinal Muscular Atrophy (SMA). Report includes an overview of trial numbers and their average enrollment in top countries conducted across the globe. The report offers coverage of disease clinical trials by region, country (G7 & E7), phase, trial status, end points status and sponsor type. Report also provides prominent drugs for in-progress trials (based on number of ongoing trials). GlobalData Clinical Trial Reports are generated using GlobalData’s proprietary database – Pharma eTrack Clinical trials database. Clinical trials are collated from 80+ different clinical trial registries, conferences, journals, news etc across the globe. Clinical trials database undergoes periodic update by dynamic process.

The report enhances the decision making capabilities and helps to create an effective counter strategies to gain competitive advantage.

Note: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

– The report provides a snapshot of the global clinical trials landscape

– Report provides top level data related to the clinical trials by Region, Country (G7 & E7), Trial Status, Trial Phase, Sponsor Type and End point status

– The report reviews top companies involved and enlists all trials (Trial title, Phase, and Status) pertaining to the company

– The report provides all the unaccomplished trials (Terminated, Suspended and Withdrawn) with reason for unaccomplishment

– The Report provides enrollment trends for the past five years

– Report provides latest news for the past three months

Note: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Reasons to buy

Assists in formulating key business strategies with regards to investment

Helps in identifying prominent locations for conducting clinical trials which saves time and cost

Provides top level analysis of Global Clinical Trials Market which helps in identifying key business opportunities

Supports understanding of trials count and enrollment trends by country in global therapeutics market

Aids in interpreting the success rates of clinical trials by providing a comparative scenario of completed and uncompleted (terminated, suspended or withdrawn) trials

Facilitates clinical trial assessment of the indication on a global, regional and country level

Note: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Companies mentioned

F. Hoffmann-La Roche Ltd

Biogen Inc

Novartis AG

PTC Therapeutics Inc

Cytokinetics Inc

Ionis Pharmaceuticals Inc

Astellas Pharma Inc

Cure SMA

PRA Health Sciences Inc

Abbott Laboratories

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 5

Report Guidance 6

GlobalData Clinical Trials Report Coverage 7

Clinical Trials by Region 8

Clinical Trials and Average Enrollment by Country 9

Top Five Countries Contributing to Clinical Trials in Asia-Pacific 12

Top Five Countries Contributing to Clinical Trials in Europe 13

Top Countries Contributing to Clinical Trials in North America 14

Top Countries Contributing to Clinical Trials in Middle East and Africa 15

Top Countries Contributing to Clinical Trials in Central and South America 16

Clinical Trials by G7 Countries: Proportion of Spinal Muscular Atrophy (SMA) to Central Nervous System Clinical Trials 17

Clinical Trials by Phase in G7 Countries 18

Clinical Trials in G7 Countries by Trial Status 19

Clinical Trials by E7 Countries: Proportion of Spinal Muscular Atrophy (SMA) to Central Nervous System Clinical Trials 20

Clinical Trials by Phase in E7 Countries 21

Clinical Trials in E7 Countries by Trial Status 22

Clinical Trials by Phase 23

In Progress Trials by Phase 24

Clinical Trials by Trial Status 25

Clinical Trials by End Point Status 26

Subjects Recruited Over a Period of Time 27

Clinical Trials by Sponsor Type 28

Prominent Sponsors 29

Top Companies Participating in Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials 31

Prominent Drugs 32

Latest Clinical Trials News on Spinal Muscular Atrophy (SMA) 33

Nov 25, 2019: Genentech gets FDA priority review status for risdiplam in SMA 33

Nov 19, 2019: Scholar Rock reports preliminary pharmacokinetic and pharmacodynamic data from TOPAZ phase 2 trial of SRK-015 for the treatment of patients with spinal muscular atrophy 33

Nov 12, 2019: Roche and PTC report positive data for risdiplam in SMA 34

Clinical Trial Profile Snapshots 35

Appendix 105

Abbreviations 105

Definitions 105

Research Methodology 106

Secondary Research 106

About GlobalData 107

Contact Us 107

Source 108

List of Tables

Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials by Region, 2020* 8

Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials and Average Enrollment by Top Countries, 2020* 10

Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials In Progress by Top Countries, 2020* 11

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Asia-Pacific, Top Five Countries, 2020* 12

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Europe, Top Five Countries, 2020* 13

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, North America, Top Countries, 2020* 14

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Middle East and Africa, Top Countries, 2020* 15

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Central and South America, Top Countries, 2020* 16

Proportion of Spinal Muscular Atrophy (SMA) to Central Nervous System Clinical Trials, G7 Countries (%), 2020* 17

Spinal Muscular Atrophy (SMA) Therapeutics, G7 Countries, Clinical Trials by Phase, 2020* 18

Spinal Muscular Atrophy (SMA) Therapeutics, G7 Countries, Clinical Trials by Trial Status, 2020* 19

Proportion of Spinal Muscular Atrophy (SMA) to Central Nervous System Clinical Trials, E7 Countries (%), 2020* 20

Spinal Muscular Atrophy (SMA) Therapeutics, E7 Countries, Clinical Trials by Phase, 2020* 21

Spinal Muscular Atrophy (SMA) Therapeutics, E7 Countries, Clinical Trials by Trial Status, 2020* 22

Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials by Phase, 2020* 23

Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials In Progress by Phase 2020* 24

Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials by Trial Status, 2020* 25

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, by End Point Status, 2020* 26

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Average Enrollment Target Trends, 2015-2019 27

Spinal Muscular Atrophy (SMA) Therapeutics Market, Global, Clinical Trials by Sponsor Type, 2020* 28

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Key Sponsors, 2020* 30

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Top Companies by Phase, 2020* 31

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Ongoing Clinical Trials by Prominent Drugs, 2020* 32

List of Figures

Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials by Region (%), 2020* 8

Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials and Average Enrollment by Top Countries, 2020* 9

Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials In Progress by Top Countries, 2020* 11

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Asia-Pacific, Top Five Countries (%), 2020* 12

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Europe, Top Five Countries (%), 2020* 13

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, North America, Top Countries (%), 2020* 14

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Middle East and Africa, Top Countries (%), 2020* 15

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Central and South America, Top Countries (%), 2020* 16

Proportion of Spinal Muscular Atrophy (SMA) to Central Nervous System Clinical Trials, G7 Countries (%), 2020* 17

Spinal Muscular Atrophy (SMA) Therapeutics, G7 Countries, Clinical Trials by Phase, 2020* 18

Spinal Muscular Atrophy (SMA) Therapeutics, G7 Countries, Clinical Trials by Trial Status, 2020* 19

Proportion of Spinal Muscular Atrophy (SMA) to Central Nervous System Clinical Trials, E7 Countries (%), 2020* 20

Spinal Muscular Atrophy (SMA) Therapeutics, E7 Countries, Clinical Trials by Phase, 2020* 21

Spinal Muscular Atrophy (SMA) Therapeutics, E7 Countries, Clinical Trials by Trial Status, 2020* 22

Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials by Phase (%), 2020* 23

Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials In Progress by Phase, 2020* 24

Spinal Muscular Atrophy (SMA) Therapeutics, Global, Clinical Trials by Trial Status, 2020* 25

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, by End Point Status, 2020* 26

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Average Enrollment Target Trends, 2015-2019 27

Spinal Muscular Atrophy (SMA) Therapeutics Market, Global, Clinical Trials by Sponsor Type (%), 2020* 28

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Key Sponsors, 2020* 29

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Top Companies by Phase, 2020* 31

Spinal Muscular Atrophy (SMA) Therapeutics Clinical Trials, Global, Ongoing Clinical Trials by Prominent Drugs, 2020* 32

GlobalData Methodology 106

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