Dystrophin (DMD) - Pipeline Review, H2 2017
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Dystrophin (DMD) - Pipeline Review, H2 2017
Summary
According to the recently published report Dystrophin (DMD) - Pipeline Review, H2 2017; Dystrophin (DMD) pipeline Target constitutes close to 27 molecules. Out of which approximately 23 molecules are developed by companies and remaining by the universities/institutes.
Dystrophin (DMD) - Dystrophin is a cytoplasmic protein. It anchors the extracellular matrix to the cytoskeleton via F-actin. It acts as ligand for dystroglycan. It act as component of the dystrophin-associated glycoprotein complex which accumulates at the neuromuscular junction and at a variety of synapses in the peripheral and central nervous systems and has a structural function in stabilizing the sarcolemma.
The report Dystrophin (DMD) - Pipeline Review, H2 2017 outlays comprehensive information on the Dystrophin (DMD) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type; that are being developed by Companies / Universities.
It also reviews key players involved in Dystrophin (DMD) targeted therapeutics development with respective active and dormant or discontinued projects. Currently, The molecules developed by companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 2, 3, 2, 5 and 10 respectively. Similarly, the universities portfolio in Phase I and Preclinical stages comprises 1 and 3 molecules, respectively. Report covers products from therapy areas Genetic Disorders and Musculoskeletal Disorders which include indications Duchenne Muscular Dystrophy and Muscular Dystrophy.
Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
Scope
- The report provides a snapshot of the global therapeutic landscape for Dystrophin (DMD)
- The report reviews Dystrophin (DMD) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved in Dystrophin (DMD) targeted therapeutics and enlists all their major and minor projects
- The report assesses Dystrophin (DMD) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type
- The report summarizes all the dormant and discontinued pipeline projects
- The report reviews latest news and deals related to Dystrophin (DMD) targeted therapeutics
Reasons to buy
- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Identify and understand the targeted therapy areas and indications for Dystrophin (DMD)
- Identify the use of drugs for target identification and drug repurposing
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies
- Plan mergers and acquisitions effectively by identifying key players and its most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Dystrophin (DMD) development landscape
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and Scope
Summary
According to the recently published report Dystrophin (DMD) - Pipeline Review, H2 2017; Dystrophin (DMD) pipeline Target constitutes close to 27 molecules. Out of which approximately 23 molecules are developed by companies and remaining by the universities/institutes.
Dystrophin (DMD) - Dystrophin is a cytoplasmic protein. It anchors the extracellular matrix to the cytoskeleton via F-actin. It acts as ligand for dystroglycan. It act as component of the dystrophin-associated glycoprotein complex which accumulates at the neuromuscular junction and at a variety of synapses in the peripheral and central nervous systems and has a structural function in stabilizing the sarcolemma.
The report Dystrophin (DMD) - Pipeline Review, H2 2017 outlays comprehensive information on the Dystrophin (DMD) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type; that are being developed by Companies / Universities.
It also reviews key players involved in Dystrophin (DMD) targeted therapeutics development with respective active and dormant or discontinued projects. Currently, The molecules developed by companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 2, 3, 2, 5 and 10 respectively. Similarly, the universities portfolio in Phase I and Preclinical stages comprises 1 and 3 molecules, respectively. Report covers products from therapy areas Genetic Disorders and Musculoskeletal Disorders which include indications Duchenne Muscular Dystrophy and Muscular Dystrophy.
Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
Scope
- The report provides a snapshot of the global therapeutic landscape for Dystrophin (DMD)
- The report reviews Dystrophin (DMD) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved in Dystrophin (DMD) targeted therapeutics and enlists all their major and minor projects
- The report assesses Dystrophin (DMD) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type
- The report summarizes all the dormant and discontinued pipeline projects
- The report reviews latest news and deals related to Dystrophin (DMD) targeted therapeutics
Reasons to buy
- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Identify and understand the targeted therapy areas and indications for Dystrophin (DMD)
- Identify the use of drugs for target identification and drug repurposing
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies
- Plan mergers and acquisitions effectively by identifying key players and its most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Dystrophin (DMD) development landscape
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and Scope
TABLE OF CONTENTS
Table of Contents
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
BioMarin Pharmaceutical Inc
Daiichi Sankyo Co Ltd
Editas Medicine Inc
Genethon SA
NS Pharma Inc
Pfizer Inc
Sarepta Therapeutics 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
casimersen - 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 DMD for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate DMD 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 Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
golodirsen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NS-065 - 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 2 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
PF-06939926 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PRO-055 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SGT-001 - 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-5051 - 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
WVE-210201 - 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 27, 2017: Sarepta Therapeutics Announces Publication of Long-Term Pulmonary Function of Eteplirsen-Treated Patients Compared to Natural History of Duchenne Muscular Dystrophy in The Journal of Neuromuscular Diseases
Nov 30, 2017: Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Patients with Duchenne Muscular Dystrophy
Nov 07, 2017: Sarepta Therapeutics Announces FDA Clearance of IND for the Companys PPMO Exon 51 Candidate, SRP-5051
Nov 06, 2017: Sarepta Therapeutics and Nationwide Childrens Hospital Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne Muscular Dystrophy
Nov 06, 2017: Wave Life Sciences Initiates Clinical Trial for Lead Program in Duchenne Muscular Dystrophy (DMD)
Sep 28, 2017: Sarepta Therapeutics to Present on Eteplirsen at the 22nd International Annual Congress of the World Muscle Society
Sep 28, 2017: Sarepta Therapeutics to Present on SRP-4053 at the 22nd International Annual Congress of the World Muscle Society
Sep 06, 2017: Sarepta Therapeutics Announces Positive Results in Its Study Evaluating Gene Expression, Dystrophin Production, and Dystrophin Localization in Patients with Duchenne Muscular Dystrophy (DMD) Amenable to Skipping Exon 53 Treated with Golodirsen (SRP-4053)
Jul 19, 2017: Sarepta Therapeutics and Clinigen Launch a Managed Access Program to Treat Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
May 11, 2017: New Preclinical Data Support SGT-001 As A Novel Treatment Approach For Duchenne Muscular Dystrophy
Apr 24, 2017: Daiichi Sankyo Announces Designation of Duchenne Muscular Dystrophy Treatment DS-5141 under SAKIGAKE Designation System
Mar 30, 2017: Sarepta Therapeutics Announces Addition of Kenneth Fischbeck, M.D. and Matthew Wood M.D., Ph.D. to the Companys Strategic and Scientific Advisory Board
Jan 06, 2017: WAVE Life Sciences Provides Update on Duchenne Muscular Dystrophy Program WVE-210201
Dec 19, 2016: Sarepta Therapeutics Announces EMA Validation of Eteplirsen Authorization Application for Treatment of Duchenne Muscular Dystrophy Amenable to Exon Skipping 51
Oct 24, 2016: Solid Biosciences Granted U.S. and E.U. Orphan Drug Designations for Lead Gene Therapy Candidate for Duchenne Muscular Dystrophy
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
Number of Products under Development by Stage of Development, H2 2017
Number of Products under Development by Therapy Areas, H2 2017
Number of Products under Development by Indication, H2 2017
Number of Products under Development by Companies, H2 2017
Products under Development by Companies, H2 2017
Products under Development by Companies, H2 2017 (Contd..1), H2 2017
Number of Products under Investigation by Universities/Institutes, H2 2017
Products under Investigation by Universities/Institutes, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Stage and Route of Administration, H2 2017
Number of Products by Stage and Molecule Type, H2 2017
Pipeline by BioMarin Pharmaceutical Inc, H2 2017
Pipeline by Daiichi Sankyo Co Ltd, H2 2017
Pipeline by Editas Medicine Inc, H2 2017
Pipeline by Genethon SA, H2 2017
Pipeline by NS Pharma Inc, H2 2017
Pipeline by Pfizer Inc, H2 2017
Pipeline by Sarepta Therapeutics Inc, H2 2017
Pipeline by WAVE Life Sciences Ltd, H2 2017
Dormant Projects, H2 2017
Discontinued Products, H2 2017
Number of Products under Development by Stage of Development, H2 2017
Number of Products under Development by Therapy Areas, H2 2017
Number of Products under Development by Indications, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Routes of Administration, H2 2017
Number of Products by Stage and Routes of Administration, H2 2017
Number of Products by Molecule Types, H2 2017
Number of Products by Stage and Molecule Types, H2 2017
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
BioMarin Pharmaceutical Inc
Daiichi Sankyo Co Ltd
Editas Medicine Inc
Genethon SA
NS Pharma Inc
Pfizer Inc
Sarepta Therapeutics 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
casimersen - 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 DMD for Duchenne Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate DMD 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 Muscular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
golodirsen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NS-065 - 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 2 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
PF-06939926 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PRO-055 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SGT-001 - 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-5051 - 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
WVE-210201 - 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 27, 2017: Sarepta Therapeutics Announces Publication of Long-Term Pulmonary Function of Eteplirsen-Treated Patients Compared to Natural History of Duchenne Muscular Dystrophy in The Journal of Neuromuscular Diseases
Nov 30, 2017: Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Patients with Duchenne Muscular Dystrophy
Nov 07, 2017: Sarepta Therapeutics Announces FDA Clearance of IND for the Companys PPMO Exon 51 Candidate, SRP-5051
Nov 06, 2017: Sarepta Therapeutics and Nationwide Childrens Hospital Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne Muscular Dystrophy
Nov 06, 2017: Wave Life Sciences Initiates Clinical Trial for Lead Program in Duchenne Muscular Dystrophy (DMD)
Sep 28, 2017: Sarepta Therapeutics to Present on Eteplirsen at the 22nd International Annual Congress of the World Muscle Society
Sep 28, 2017: Sarepta Therapeutics to Present on SRP-4053 at the 22nd International Annual Congress of the World Muscle Society
Sep 06, 2017: Sarepta Therapeutics Announces Positive Results in Its Study Evaluating Gene Expression, Dystrophin Production, and Dystrophin Localization in Patients with Duchenne Muscular Dystrophy (DMD) Amenable to Skipping Exon 53 Treated with Golodirsen (SRP-4053)
Jul 19, 2017: Sarepta Therapeutics and Clinigen Launch a Managed Access Program to Treat Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
May 11, 2017: New Preclinical Data Support SGT-001 As A Novel Treatment Approach For Duchenne Muscular Dystrophy
Apr 24, 2017: Daiichi Sankyo Announces Designation of Duchenne Muscular Dystrophy Treatment DS-5141 under SAKIGAKE Designation System
Mar 30, 2017: Sarepta Therapeutics Announces Addition of Kenneth Fischbeck, M.D. and Matthew Wood M.D., Ph.D. to the Companys Strategic and Scientific Advisory Board
Jan 06, 2017: WAVE Life Sciences Provides Update on Duchenne Muscular Dystrophy Program WVE-210201
Dec 19, 2016: Sarepta Therapeutics Announces EMA Validation of Eteplirsen Authorization Application for Treatment of Duchenne Muscular Dystrophy Amenable to Exon Skipping 51
Oct 24, 2016: Solid Biosciences Granted U.S. and E.U. Orphan Drug Designations for Lead Gene Therapy Candidate for Duchenne Muscular Dystrophy
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List Of Tables
Number of Products under Development by Stage of Development, H2 2017
Number of Products under Development by Therapy Areas, H2 2017
Number of Products under Development by Indication, H2 2017
Number of Products under Development by Companies, H2 2017
Products under Development by Companies, H2 2017
Products under Development by Companies, H2 2017 (Contd..1), H2 2017
Number of Products under Investigation by Universities/Institutes, H2 2017
Products under Investigation by Universities/Institutes, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Stage and Route of Administration, H2 2017
Number of Products by Stage and Molecule Type, H2 2017
Pipeline by BioMarin Pharmaceutical Inc, H2 2017
Pipeline by Daiichi Sankyo Co Ltd, H2 2017
Pipeline by Editas Medicine Inc, H2 2017
Pipeline by Genethon SA, H2 2017
Pipeline by NS Pharma Inc, H2 2017
Pipeline by Pfizer Inc, H2 2017
Pipeline by Sarepta Therapeutics Inc, H2 2017
Pipeline by WAVE Life Sciences Ltd, H2 2017
Dormant Projects, H2 2017
Discontinued Products, H2 2017
List Of Figures
Number of Products under Development by Stage of Development, H2 2017
Number of Products under Development by Therapy Areas, H2 2017
Number of Products under Development by Indications, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Routes of Administration, H2 2017
Number of Products by Stage and Routes of Administration, H2 2017
Number of Products by Molecule Types, H2 2017
Number of Products by Stage and Molecule Types, H2 2017
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