Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Dystrophin (DMD) - Overview
Dystrophin (DMD) - Therapeutics Development
Products under Development by Stage of Development
Products under Development by Therapy Area
Products under Development by Indication
Products under Development by Companies
Products under Development by Universities/Institutes
Dystrophin (DMD) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Dystrophin (DMD) - Companies Involved in Therapeutics Development
Alpha Anomeric
Audentes Therapeutics Inc
Daiichi Sankyo Co Ltd
Dystrogen Therapeutics SA
Editas Medicine Inc
Evox Therapeutics Ltd
MyoGene Bio LLC
Nippon Shinyaku Co Ltd
NS Pharma Inc
OliPass Corporation
Pepgen Ltd
Pfizer Inc
Sarepta Therapeutics Inc
Spilcyte Llc
Sutura Therapeutics Ltd
Tolerion Inc
Vertex Pharmaceuticals Inc
Wave Life Sciences Ltd
Dystrophin (DMD) - Drug Profiles
Antisense Oligonucleotide 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Antisense Oligonucleotides to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AT-702 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AT-751 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AT-753 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
casimersen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CYMD-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DMD-1742 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DS-5141 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
eteplirsen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy 1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
golodirsen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NS-089 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides 3 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides 5 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides 6 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PF-06939926 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Protein to Target Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-4008 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-4044 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-4050 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-4052 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-4055 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-5044 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-5045 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-5050 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-5051 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-5052 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-5053 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRP-9001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy 2 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SUT-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
viltolarsen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Dystrophin (DMD) - Dormant Products
Dystrophin (DMD) - Discontinued Products
Dystrophin (DMD) - Product Development Milestones
Featured News & Press Releases
Dec 13, 2019: Sarepta?s DMD therapy Vyondys 53 secures FDA accelerated approval
Nov 05, 2019: Dystrogen Therapeutics announces that treatment with Dystrophin expressing Chimeric (DEC) Cells improves cardiac function in Preclinical Duchenne’s Study
Aug 21, 2019: Sarepta fails to receive FDA approval for DMD drug golodirsen
Aug 08, 2019: Sarepta Therapeutics comments on erroneous submission to US FDA Adverse Event Reporting System (FAERS)
Jul 09, 2019: New alysis shows drug slows down respiratory decline
Jul 01, 2019: AskBio’s Perspective on Pfizer’s 1b clinical trial results on Duchenne MD Gene
Jun 28, 2019: Pfizer presents initial clinical data on phase 1b gene therapy study for Duchenne Muscular Dystrophy (DMD)
Apr 18, 2019: Parent Project Muscular Dystrophy awards $100,000 grant to tionwide Children’s Hospital to further explore GALGT2 gene therapy in duchenne muscular dystrophy
Mar 28, 2019: Sarepta Therapeutics announces positive expression results from the Casimersen (SRP-4045) arm of the ESSENCE study
Mar 25, 2019: Sarepta Therapeutics to provide update on Duchenne Muscular Dystrophy gene therapy program
Feb 15, 2019: FDA accepts Sarepta?s NDA for precision medicine Golodirsen
Feb 06, 2019: NS Pharma begins rolling NDA submission to FDA for Viltolarsen (NS-065/NCNP-01)
Dec 20, 2018: Sarepta Therapeutics completes Submission of New Drug Application Seeking approval of Golodirsen (SRP-4053) in patients with Duchenne Muscular Dystrophy Ameble to Skipping Exon 53
Nov 13, 2018: Evox Therapeutics wins funding from Duchenne UK to explore exosome-mediated delivery of dystrophin
Oct 03, 2018: NS-065/NCNP-01 (Viltolarsen) of Nippon Shinyaku? in-house product presentation on results of Phase I/II study in Japan
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures


Number of Products under Development by Stage of Development, H2 2019
Number of Products under Development by Therapy Areas, H2 2019
Number of Products under Development by Top 10 Indications, H2 2019
Number of Products by Stage and Mechanism of Actions, H2 2019
Number of Products by Routes of Administration, H2 2019
Number of Products by Stage and Routes of Administration, H2 2019
Number of Products by Molecule Types, H2 2019
Number of Products by Stage and Molecule Types, H2 2019


List of Tables


Number of Products under Development by Stage of Development, H2 2019
Number of Products under Development by Therapy Areas, H2 2019
Number of Products under Development by Indication, H2 2019
Number of Products under Development by Companies, H2 2019
Products under Development by Companies, H2 2019
Products under Development by Companies, H2 2019 (Contd..1), H2 2019
Products under Development by Companies, H2 2019 (Contd..2), H2 2019
Number of Products under Investigation by Universities/Institutes, H2 2019
Products under Investigation by Universities/Institutes, H2 2019
Number of Products by Stage and Mechanism of Actions, H2 2019
Number of Products by Stage and Route of Administration, H2 2019
Number of Products by Stage and Molecule Type, H2 2019
Pipeline by Alpha Anomeric, H2 2019
Pipeline by Audentes Therapeutics Inc, H2 2019
Pipeline by Daiichi Sankyo Co Ltd, H2 2019
Pipeline by Dystrogen Therapeutics SA, H2 2019
Pipeline by Editas Medicine Inc, H2 2019
Pipeline by Evox Therapeutics Ltd, H2 2019
Pipeline by MyoGene Bio LLC, H2 2019
Pipeline by Nippon Shinyaku Co Ltd, H2 2019
Pipeline by NS Pharma Inc, H2 2019
Pipeline by OliPass Corporation, H2 2019
Pipeline by Pepgen Ltd, H2 2019
Pipeline by Pfizer Inc, H2 2019
Pipeline by Sarepta Therapeutics Inc, H2 2019
Pipeline by Spinalcyte Llc, H2 2019
Pipeline by Sutura Therapeutics Ltd, H2 2019
Pipeline by Tolerion Inc, H2 2019
Pipeline by Vertex Pharmaceuticals Inc, H2 2019
Pipeline by Wave Life Sciences Ltd, H2 2019
Dormant Projects, H2 2019
Discontinued Products, H2 2019