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Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2018

Published: August 2018
Pages: 128
Code: 0F715187D48409AC7380AB44ECABEFB0
Format: PDF
Publisher Global Markets Direct
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Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide , provides an overview of the Spinal Muscular Atrophy (SMA) (Central Nervous System) pipeline landscape.
Spinal muscular atrophy (SMA) is a genetic disease that attacks nerve cells, called motor neurons, in the spinal cord. These critically important cells are responsible for supplying electrical and chemical messages to muscle cells. Without the proper input from the motor neurons, muscle cells cannot function properly. The muscle cells will, therefore, become much smaller (atrophy) and will produce symptoms of muscle weakness. This can affect walking, crawling, breathing, swallowing, and head and neck control.
Report Highlights
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide , provides comprehensive information on the therapeutics under development for Spinal Muscular Atrophy (SMA) (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.
The Spinal Muscular Atrophy (SMA) (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Spinal Muscular Atrophy (SMA) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 4, 2, 4, 20 and 7 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 7 and 3 molecules, respectively.
Spinal Muscular Atrophy (SMA) (Central Nervous System) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Additonal Information

  • - The pipeline guide provides a snapshot of the global therapeutic landscape of Spinal Muscular Atrophy (SMA) (Central Nervous System).
  • - The pipeline guide reviews pipeline therapeutics for Spinal Muscular Atrophy (SMA) (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • - The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • - The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • - The pipeline guide reviews key companies involved in Spinal Muscular Atrophy (SMA) (Central Nervous System) therapeutics and enlists all their major and minor projects.
  • - The pipeline guide evaluates Spinal Muscular Atrophy (SMA) (Central Nervous System) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • - The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • - The pipeline guide reviews latest news related to pipeline therapeutics for Spinal Muscular Atrophy (SMA) (Central Nervous System)

Companies Mentioned

AndroScience Corp
AveXis Inc
Biogen Inc
BioMarin Pharmaceutical Inc
Cytokinetics Inc
Exicure Inc
F. Hoffmann-La Roche Ltd
Genethon SA
Ionis Pharmaceuticals Inc
Kowa Co Ltd
Neurodyn Life Sciences Inc
Neurotune AG
Novartis AG
Ono Pharmaceutical Co Ltd
Paratek Pharmaceuticals Inc
Recursion Pharmaceuticals Inc
Sarepta Therapeutics Inc
Scholar Rock Inc
Spotlight Innovation Inc
Vybion Inc

Reasons to buy

Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies. Recognize emerging players with potentially strong product portfolio and create effective counterstrategies to gain competitive advantage. Find and recognize significant and varied types of therapeutics under development for Spinal Muscular Atrophy (SMA) (Central Nervous System). Classify potential new clients or partners in the target demographic. Develop tactical initiatives by understanding the focus areas of leading companies. Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics. Formulate corrective measures for pipeline projects by understanding Spinal Muscular Atrophy (SMA) (Central Nervous System) pipeline depth and focus of Indication therapeutics. Develop and design inlicensing and outlicensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope. Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the knowhow what drove them from pipeline.
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  • Table of Contents
  • List of Tables
  • List of Figures
  • Introduction
  • Global Markets Direct Report Coverage
  • Spinal Muscular Atrophy (SMA) - Overview
  • Spinal Muscular Atrophy (SMA) - Therapeutics Development
  • Pipeline Overview
  • Pipeline by Companies
  • Pipeline by Universities/Institutes
  • Products under Development by Companies
  • Products under Development by Universities/Institutes
  • Spinal Muscular Atrophy (SMA) - Therapeutics Assessment
  • Assessment by Target
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
  • Spinal Muscular Atrophy (SMA) - Companies Involved in Therapeutics Development
  • AndroScience Corp
  • AveXis Inc
  • Biogen Inc
  • BioMarin Pharmaceutical Inc
  • Cytokinetics Inc
  • Exicure Inc
  • F. Hoffmann-La Roche Ltd
  • Genethon SA
  • Ionis Pharmaceuticals Inc
  • Kowa Co Ltd
  • Neurodyn Life Sciences Inc
  • Neurotune AG
  • Novartis AG
  • Ono Pharmaceutical Co Ltd
  • Paratek Pharmaceuticals Inc
  • Recursion Pharmaceuticals Inc
  • Sarepta Therapeutics Inc
  • Scholar Rock Inc
  • Spotlight Innovation Inc
  • Vybion Inc
  • Spinal Muscular Atrophy (SMA) - Drug Profiles
  • AAD-2004 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ALB-111 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ALG-802 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • amifampridine phosphate - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Antisense Oligonucleotides for Spinal Muscular Atrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Antisense Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ARM-210 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ASC-J9/ASC-JM.X1 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ASCJ-9 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • BIIB-110 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • branaplam - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • INT-41 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • LDN-5178 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • maresin-1 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ND-602 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • NT-1654 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • onasemnogene abeparvovec - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • PMO-25 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • PTKSMA-1 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • REC-0000716 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • REC-0001202 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • reldesemtiv - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • risdiplam - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Rycal - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecule to Inhibit GSK-3 for Spinal Muscular Atrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules 2 to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules for Central Nervous System Disorders - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules for Kennedy's Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules for Spinal Muscular Atrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules for X-Linked Infantile Spinal Muscular Atrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules to Inhibit SRSF10 and BCLAF1 for HIV Infection, Spinal Muscular Atrophy and Colon Cancer - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SMN-2 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SRK-105 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • STL-182 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • TEC-1 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • valproate sodium - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Xcel-hNu - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Xcel-hNuP - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Spinal Muscular Atrophy (SMA) - Dormant Projects
  • Spinal Muscular Atrophy (SMA) - Discontinued Products
  • Spinal Muscular Atrophy (SMA) - Product Development Milestones
  • Featured News & Press Releases
  • Jul 24, 2018: Spotlight Innovation spinal muscular atrophy research collaborator professor Kevin Hodgetts receives $300,000 grant from Cure SMA
  • 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
  • Jun 14, 2018: Exicure to Present Data at the Cure Spinal Muscular Atrophy Annual Conference in Dallas
  • 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
  • May 31, 2018: Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015
  • May 08, 2018: AveXis Issues Community Statement on the SPR1NT Trial
  • Apr 25, 2018: AveXis Announces First Patient Dosed in Phase 3 Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3
  • Apr 24, 2018: AveXis Presents Initial Data from Pivotal U.S. Trial for SMA Type 1 and 24-Month Follow-Up Data from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology
  • Apr 24, 2018: Cytokinetics Announces Presentation of Data From Phase 2 Clinical Study of Reldesemtiv in Patients With Spinal Muscular Atrophy at the 2018 Annual Cure SMA Conference
  • Apr 19, 2018: AveXis to Present AVXS-101 Data at the Annual Meeting of the American Academy of Neurology
  • Mar 29, 2018: Scholar Rock Granted Orphan Drug Designation by the FDA for SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy
  • Mar 27, 2018: AveXis Gene Therapy Awarded SAKIGAKE Designation for Spinal Muscular Atrophy Type 1
  • Mar 13, 2018: Newly Issued U.S. Patent Covers Spotlight Innovations Spinal Muscular Atrophy Drug Candidates
  • Feb 21, 2018: Phase 2 Clinical Trial Results for CK-2127107 Expected in Second Quarter of 2018
  • Feb 02, 2018: Scholar Rock Announces Publication of Comprehensive Pharmacological Study of SRK-015, a Novel Inhibitor of Myostatin Activation, Supporting the Treatment of Muscle Atrophy Disorders
  • Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact Us
  • Disclaimer
  • List of Tables
  • Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2018
  • Number of Products under Development by Companies, H2 2018
  • Number of Products under Development by Companies, H2 2018 (Contd..1), H2 2018
  • Number of Products under Development by Universities/Institutes, H2 2018
  • Products under Development by Companies, H2 2018
  • Products under Development by Companies, H2 2018 (Contd..1), H2 2018
  • Products under Development by Companies, H2 2018 (Contd..2), H2 2018
  • Products under Development by Universities/Institutes, H2 2018
  • Number of Products by Stage and Target, H2 2018
  • Number of Products by Stage and Mechanism of Action, H2 2018
  • Number of Products by Stage and Route of Administration, H2 2018
  • Number of Products by Stage and Molecule Type, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by AndroScience Corp, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by AveXis Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Biogen Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by BioMarin Pharmaceutical Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Exicure Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Genethon SA, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Ionis Pharmaceuticals Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Kowa Co Ltd, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Neurodyn Life Sciences Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Ono Pharmaceutical Co Ltd, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Paratek Pharmaceuticals Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Recursion Pharmaceuticals Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Scholar Rock Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Spotlight Innovation Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Pipeline by Vybion Inc, H2 2018
  • Spinal Muscular Atrophy (SMA) - Dormant Projects, H2 2018
  • Spinal Muscular Atrophy (SMA) - Dormant Projects, H2 2018 (Contd..1), H2 2018
  • Spinal Muscular Atrophy (SMA) - Discontinued Products, H2 2018
  • List of Figures
  • Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2018
  • Number of Products under Development by Companies, H2 2018
  • Number of Products under Development by Universities/Institutes, H2 2018
  • Number of Products by Top 10 Targets, H2 2018
  • Number of Products by Stage and Top 10 Targets, H2 2018
  • Number of Products by Top 10 Mechanism of Actions, H2 2018
  • Number of Products by Stage and Top 10 Mechanism of Actions, H2 2018
  • Number of Products by Routes of Administration, H2 2018
  • Number of Products by Stage and Routes of Administration, H2 2018
  • Number of Products by Molecule Types, H2 2018
  • Number of Products by Stage and Molecule Types, H2 2018

Keywords
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