Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2018

Publisher Name :
Date: 20-Feb-2018
No. of pages: 113
Inquire Before Buying

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2018, 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 Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2018, 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, 3, 1, 13 and 3 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 7 and 2 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.

Scope

  • 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)

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 counter-strategies 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 in-licensing and out-licensing 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 know-how what drove them from pipeline.

Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2018

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
AveXis Inc
BioMarin Pharmaceutical Inc
Cytokinetics Inc
Exicure Inc
F. Hoffmann-La Roche Ltd
Genethon SA
Genzyme Corp
Kowa Co Ltd
Neurotune AG
Novartis AG
Paratek Pharmaceuticals Inc
Recursion Pharmaceuticals Inc
Sarepta Therapeutics Inc
Scholar Rock Inc
Spotlight Innovation Inc
Voyager Therapeutics 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
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
AVXS-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
branaplam - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CK-2127107 - 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
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
K-828SP - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NT-1654 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
olesoxime - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides for Spinal Muscular Atrophy and Huntington Disease - 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
RG-7916 - 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 for Central Nervous System Disorders - 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 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 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
SRK-105 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
STL-182 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VYSMN-101 - 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
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
Jan 30, 2018: AveXis to Initiate Screening for Remaining Patients in Pivotal Trial of AVXS-101 for SMA Type 1 Following Review of Preliminary Data from First Three Patients
Jan 27, 2018: Preliminary Data from FIREFISH trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy
Jan 23, 2018: Data from RG7916 Programs in SMA to be Presented at the International Scientific Congress on Spinal Muscular Atrophy
Jan 23, 2018: Scholar Rock and Collaborators at Harvard Medical School and Northeastern University Elucidate Molecular Basis of Myostatin Activation - a Key Physiological Process in Muscle Health
Jan 16, 2018: AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy
Jan 04, 2018: AveXis Announces Alignment with FDA on Next Steps Toward a BLA Submission for AVXS-101 in SMA Type 1
Jan 03, 2018: Cytokinetics Provides on CK-2127107
Dec 13, 2017: AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101
Dec 11, 2017: Cytokinetics Publishes Clinical Trial Data for CK-2127107
Nov 21, 2017: Catalyst Pharmaceuticals Announces Phase 2 Study of Firdapse in Ambulatory Patients with Spinal Muscular Atrophy (SMA)
Nov 01, 2017: AveXis Announces New England Journal of Medicine Publication of Phase 1 Data of AVXS-101 Gene Replacement Therapy in Spinal Muscular Atrophy Type 1
Oct 12, 2017: Spinal Muscular Atrophy Program Advances into Pivotal Study in SMA Patients
Oct 03, 2017: AveXis to Report Top-line Data from the Phase 1 Clinical Trial of AVXS-101 in SMA Type 1 at the International Annual Congress of the World Muscle Society
Oct 03, 2017: RG7916 Increased SMN Protein Production in SUNFISH Clinical Trial in Patients with Type 2/3 Spinal Muscular Atrophy
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), H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Companies, H1 2018 (Contd..1), H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018 (Contd..1), H1 2018
Products under Development by Universities/Institutes, H1 2018
Number of Products by Stage and Target, H1 2018
Number of Products by Stage and Mechanism of Action, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by AveXis Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by BioMarin Pharmaceutical Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Exicure Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Genethon SA, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corp, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Kowa Co Ltd, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Paratek Pharmaceuticals Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Recursion Pharmaceuticals Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Scholar Rock Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Spotlight Innovation Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Voyager Therapeutics Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2018
Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2018 (Contd..1), H1 2018
Spinal Muscular Atrophy (SMA) - Discontinued Products, H1 2018

List of Figures

Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Number of Products by Top 10 Targets, H1 2018
Number of Products by Stage and Top 10 Targets, H1 2018
Number of Products by Top 10 Mechanism of Actions, H1 2018
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
  • Global Vasculitis Treatment Market 2018 by Manufacturers, Regions, Type and Application, Forecast to 2023
    Published: 25-Sep-2018        Price: US 3480 Onwards        Pages: 136
    Vasculitis is a general term that refers to inflammation of blood vessels. When blood vessels become inflamed, they may become weakened, stretch, and either increase in size or become narrow -- even to the point of closing entirely.- Vasculitis can affect people of all ages, but there are types of vasculitis that occur in certain age groups more often than others. Scope of the Report: This report focuses on the Vasculitis Treatment in global market, especially in No......
  • Global Light Therapy Market 2018 by Manufacturers, Countries, Type and Application, Forecast to 2023
    Published: 24-Sep-2018        Price: US 3480 Onwards        Pages: 115
    Light therapy consists of exposure to daylight or to specific wavelengths of light using polychromatic polarised light, lasers, light-emitting diodes, fluorescent lamps, dichroic lamps or very bright, full-spectrum light. The light is administered for a prescribed amount of time and, in some cases, at a specific time of day. Scope of the Report: This report studies the Light Therapy market status and outlook of Global and major regions, from angles of players, countries, pr......
  • Global Thyroid Gland Disorder Treatment Market 2018 by Manufacturers, Countries, Type and Application, Forecast to 2023
    Published: 19-Sep-2018        Price: US 3480 Onwards        Pages: 123
    Hormones released by thyroid gland play a crucial role in stimulating metabolism, growth, and calories processing. Globally, thyroid gland disorders such as hypothyroidism, hyperthyroidism, and thyroid nodules affect around 5% to 10% of the population. Hyperthyroidism and hypothyroidism affect majority of the population primarily due to growing incidence of autoimmune diseases such as Grave's disease and Hashimoto's thyroiditis. Moreover, iodine deficiency is a widespread problem across the glob......
  • 2018-2023 Global Enzyme Replacement Therapy Market Report (Status and Outlook)
    Published: 18-Sep-2018        Price: US 4660 Onwards        Pages: 132
    In this report, LP Information studies the present scenario (with the base year being 2017) and the growth prospects of global Enzyme Replacement Therapy market for 2018-2023. Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. Usually, this is done by giving the patient an intravenous (IV) infusion of a solution containing the enzyme. The classification of enzyme replacement therapy includes injectable a......
  • Global Varicose Vein Treatment Market Insights, Forecast to 2025
    Published: 18-Sep-2018        Price: US 3900 Onwards        Pages: 124
    This report studies the global market size of Varicose Vein Treatment in key regions like North America, Europe, Asia Pacific, Central & South America and Middle East & Africa, focuses on the consumption of Varicose Vein Treatment in these regions. This research report categorizes the global Varicose Vein Treatment market by players/brands, region, type and application. This report also studies the global market status, competition landscape, market share, growth rate, future trends, mark......
  • 2018-2023 Global Digital Therapeutics & Wellness Market Report (Status and Outlook)
    Published: 18-Sep-2018        Price: US 4660 Onwards        Pages: 157
    In this report, LP Information studies the present scenario (with the base year being 2017) and the growth prospects of global Digital Therapeutics & Wellness market for 2018-2023. Digital Therapeutics is an innovative new class of medicine that gives participants access to the world's most effective behavior therapies, enhanced with smart technology, and delivered directly to their fingerprints or front door. Digital therapeutics may be up and coming, but they also have a ......
  • Graft Versus Host Disease (GVHD) - Pipeline Review, H2 2018
    Published: 18-Sep-2018        Price: US 2000 Onwards        Pages: 498
    Graft Versus Host Disease (GVHD) - Pipeline Review, H2 2018 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Graft Versus Host Disease (GVHD) - Pipeline Review, H2 2018, provides an overview of the Graft Versus Host Disease (GVHD) (Immunology) pipeline landscape. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted donor ......
  • Psoriasis - Pipeline Review, H2 2018
    Published: 18-Sep-2018        Price: US 2500 Onwards        Pages: 726
    Psoriasis - Pipeline Review, H2 2018 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Psoriasis - Pipeline Review, H2 2018, provides an overview of the Psoriasis (Immunology) pipeline landscape. Psoriasis is a chronic skin condition caused by an overactive immune system. Symptoms of the disease include shedding of cells, flared up skin, itchiness, and burning sensation, swollen and stiff joints. Predisposing ......
  • 2018-2023 Global Infantile Spasms Therapeutics Consumption Market Report
    Published: 18-Sep-2018        Price: US 4660 Onwards        Pages: 130
    In this report, LP Information covers the present scenario (with the base year being 2017) and the growth prospects of global Infantile Spasms Therapeutics market for 2018-2023. An infantile spasm (IS), also known as West Syndrome, is a specific type of seizure seen in an epilepsy syndrome of infancy and childhood. West Syndrome is characterized by infantile spasms, developmental regression, and a specific pattern on electroencephalography (EEG) testing called hypsarrhythmia (chao......
  • SERVICES
    Value for Money
    We believe in "optimum utilization of available budget and resources". While servicing our clients' (your) market research requirements, we keep the same approach in focus to help you get the best value for your $$s.
    Ever Growing Inventory
    Ranging from the smallest feasible / required data (datasheets, data facts, SWOT analysis, company profiles, etc) to full research reports that help you make decisions, our inventory is updated almost on a daily basis with the latest industry reports from domain experts that track more than 5000 niche sectors.
    One Stop Solution
    Need a custom research report on medical devices market? Require all available business intelligence on 3D printing industry? Exploring F&B sector of a particular country/region? RnRMarketResearch.com is your one-stop-solution to all market intelligence needs. We not only offer custom research and consulting services, we also "bundle" reports to meet your needs and help you fetch the data analysis you require for your business.
    Dedicated Client Engagement
    Not limited to only "finding" relevant reports for you, our client engagement team dedicates its efforts to understand your "business need" and accordingly maps available research data to help you move forward. Call "your" client engagement executive any time of your day and get your questions answered in order to make the correct business decision.
    Saving Time and Efforts
    Simply share your research requirement details with us and let us do all the hard work to find required intelligence for you. When you add up our "one stop solution" and "dedicated client engagement" services mentioned above, you obviously know the time and effort saving you do by working with us.
    Payment Flexibility
    Working with Fortune 500 organizations, we understand the importance of being flexible for payments. Share your payment terms with us and we will surely match up to them to ensure you get access to required business intelligence data without having to wait for the payment to be done.
    Post-Purchase Research Support
    Have questions after reading a report / datasheet bought through us? Not sure about the methodology used for data available in the research? Talk to us / Share your questions with us and if required, we will connect you with the analyst(s)/author(s) of the report(s) and ensure you get satisfactory answers for the same. Need more data / analysis / report(s) on the topic of your research/project? The RnRMarketResearch.com team is here for you 24X7 to support you with your post-purchase requirements. Subscription Offers & Packages (Get in touch with us for more details - sales@rnrmarketresearch.com / +1 888 391 5441 )
    • Ad Hoc
    • Pay - as - you - go / Bucket Subscriptions
    • Fixed Cost for #of reports
    • Customize / Personalize as per your needs