Pompe Disease - Pipeline Review, H1 2017

Publisher Name :
Date: 16-May-2017
No. of pages: 52
Inquire Before Buying

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Pompe Disease - Pipeline Review, H1 2017, provides an overview of the Pompe Disease (Metabolic Disorders) pipeline landscape.

Pompe disease is an inherited disorder caused by defect in a gene called GAA. The GAA gene is responsible for the production of the GAA enzyme (acid alpha-glucosidase). This enzyme is needed to break down glycogen, a form of sugar stored in muscle cells. When too much glycogen builds up in the muscle cells, the cells become damaged and the muscles cannot function properly. Symptoms include respiratory failure, muscle pain, proximal muscle weakness, respiratory tract infections, headache and difficulty chewing or jaw muscle fatigue.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Pompe Disease - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Pompe Disease (Metabolic Disorders), 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 Pompe Disease (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Pompe Disease 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, IND/CTA Filed, Preclinical and Discovery stages are 1, 1, 1, 8 and 2 respectively. Similarly, the Universities portfolio in Phase II and Preclinical stages comprises 1 and 2 molecules, respectively.

Pompe Disease (Metabolic Disorders) 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 Pompe Disease (Metabolic Disorders).

  • The pipeline guide reviews pipeline therapeutics for Pompe Disease (Metabolic Disorders) 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 Pompe Disease (Metabolic Disorders) therapeutics and enlists all their major and minor projects.

  • The pipeline guide evaluates Pompe Disease (Metabolic Disorders) 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 Pompe Disease (Metabolic Disorders)

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 Pompe Disease (Metabolic Disorders).

  • 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 Pompe Disease (Metabolic Disorders) 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.

Pompe Disease - Pipeline Review, H1 2017

Table of Contents

List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Pompe Disease - Overview
Pompe Disease - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Pompe Disease - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Pompe Disease - Companies Involved in Therapeutics Development
Amicus Therapeutics Inc
Audentes Therapeutics Inc
EpiVax Inc
Etubics Corp
Genzyme Corp
greenovation Biotech GmbH
Huons Co Ltd
JCR Pharmaceuticals Co Ltd
Oxyrane Belgium NV
Pharming Group NV
Sarepta Therapeutics Inc
Pompe Disease - Drug Profiles
Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AT-982 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ATB-200 + miglustat - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
GZ-402666 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
HU-023 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JR-162 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
MOSS-GAA - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OXY-2810 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PGN-004 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Alpha Glucosidase Replacement for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Synthetic Peptides for Pompe disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VAL-1221 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Pompe Disease - Dormant Projects
Pompe Disease - Discontinued Products
Pompe Disease - Product Development Milestones
Featured News & Press Releases
Mar 03, 2017: Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease
Nov 17, 2016: Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease to Begin in the UK
Nov 04, 2016: Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease
Mar 03, 2016: Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease
Sep 03, 2015: Valerion Therapeutics Announces Positive Results Of Non-Clinical Research Study at SSIEM Annual Symposium
May 11, 2015: Audentes Therapeutics To Present New Data On AT-002 at American Society of Gene and Cell Therapy Annual Meeting
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables

Number of Products under Development for Pompe Disease, H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products under Development by Universities/Institutes, H1 2017
Products under Development by Companies, H1 2017
Products under Development by Universities/Institutes, H1 2017
Number of Products by Stage and Target, H1 2017
Number of Products by Stage and Mechanism of Action, H1 2017
Number of Products by Stage and Route of Administration, H1 2017
Number of Products by Stage and Molecule Type, H1 2017
Pompe Disease - Pipeline by Amicus Therapeutics Inc, H1 2017
Pompe Disease - Pipeline by Audentes Therapeutics Inc, H1 2017
Pompe Disease - Pipeline by EpiVax Inc, H1 2017
Pompe Disease - Pipeline by Etubics Corp, H1 2017
Pompe Disease - Pipeline by Genzyme Corp, H1 2017
Pompe Disease - Pipeline by greenovation Biotech GmbH, H1 2017
Pompe Disease - Pipeline by Huons Co Ltd, H1 2017
Pompe Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2017
Pompe Disease - Pipeline by Oxyrane Belgium NV, H1 2017
Pompe Disease - Pipeline by Pharming Group NV, H1 2017
Pompe Disease - Pipeline by Sarepta Therapeutics Inc, H1 2017
Pompe Disease - Dormant Projects, H1 2017
Pompe Disease - Discontinued Products, H1 2017

List of Figures

Number of Products under Development for Pompe Disease, H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products under Development by Universities/Institutes, H1 2017
Number of Products by Stage and Top 10 Targets, H1 2017
Number of Products by Mechanism of Actions, H1 2017
Number of Products by Stage and Mechanism of Actions, H1 2017
Number of Products by Routes of Administration, H1 2017
Number of Products by Stage and Routes of Administration, H1 2017
Number of Products by Molecule Types, H1 2017
Number of Products by Stage and Molecule Types, H1 2017
  • Global Peptide Therapeutics Market Professional Survey Report 2017
    Published: 12-Sep-2017        Price: US 3500 Onwards        Pages: 107
    This report studies Peptide Therapeutics in Global market, especially in North America, China, Europe, Southeast Asia, Japan and India, with production, revenue, consumption, import and export in these regions, from 2012 to 2016, and forecast to 2022. This report focuses on top manufacturers in global market, with production, price, revenue and market share for each manufacturer, covering - Pfizer - Eli Lilly and Company - Bachem Holding - Amgen ......
  • Pseudomonas aeruginosa Pneumonia - Pipeline Review, H2 2017
    Published: 12-Sep-2017        Price: US 2000 Onwards        Pages: 32
    Pseudomonas aeruginosa Pneumonia - Pipeline Review, H2 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Pseudomonas aeruginosa Pneumonia - Pipeline Review, H2 2017, provides an overview of the Pseudomonas aeruginosa Pneumonia (Infectious Disease) pipeline landscape. Pseudomonas aeruginosa has become an important cause of pneumonia. It is the most common pathogen isolated from patients who have been hospita......
  • Achondroplasia - Pipeline Review, H2 2017
    Published: 12-Sep-2017        Price: US 2000 Onwards        Pages: 36
    Achondroplasia - Pipeline Review, H2 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Achondroplasia - Pipeline Review, H2 2017, provides an overview of the Achondroplasia (Musculoskeletal Disorders) pipeline landscape. Achondroplasia is a bone growth disorder that causes disproportionate dwarfism. This is caused by mutations in the FGFR3 gene. Symptoms include decreased muscle tone, apnea, hydrocephalus, ......
  • Dupuytren Contracture - Pipeline Review, H2 2017
    Published: 12-Sep-2017        Price: US 2000 Onwards        Pages: 31
    Dupuytren Contracture - Pipeline Review, H2 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Dupuytren Contracture - Pipeline Review, H2 2017, provides an overview of the Dupuytren Contracture (Musculoskeletal Disorders) pipeline landscape. Dupuytren contracture is a hand deformity that usually develops slowly, over decades. Dupuytren contracture affects the connective tissue under the skin of the palm. It......
  • Fibrosis - Pipeline Review, H2 2017
    Published: 12-Sep-2017        Price: US 2000 Onwards        Pages: 122
    Fibrosis - Pipeline Review, H2 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fibrosis - Pipeline Review, H2 2017, provides an overview of the Fibrosis (Musculoskeletal Disorders) pipeline landscape. Fibrosis is the excessive formation of fibrous bands of scar tissue in between muscle fibers. Fibrous scar tissue develops after the muscle has been damaged to fill in the open spaces in the injured muscle, ......
  • Anesthetic Effect - Pipeline Review, H2 2017
    Published: 12-Sep-2017        Price: US 2000 Onwards        Pages: 88
    Anesthetic Effect - Pipeline Review, H2 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Anesthetic Effect - Pipeline Review, H2 2017, provides an overview of the Anesthetic Effect (Central Nervous System) pipeline landscape. Anesthetic effect causes a loss of consciousness. The factors that can increase risk of complications include smoking, obstructive sleep apnea, obesity, high blood pressure, history o......
  • Neuromyelitis Optica (Devic's Syndrome) - Pipeline Review, H2 2017
    Published: 12-Sep-2017        Price: US 2000 Onwards        Pages: 92
    Neuromyelitis Optica (Devic's Syndrome) - Pipeline Review, H2 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Neuromyelitis Optica (Devic's Syndrome) - Pipeline Review, H2 2017, provides an overview of the Neuromyelitis Optica (Devic's Syndrome) (Central Nervous System) pipeline landscape. Neuromyelitis optica (NMO), also known as Devic's disease, is an uncommon neurological condition caused by disease or......
  • Friedreich Ataxia - Pipeline Review, H2 2017
    Published: 12-Sep-2017        Price: US 2000 Onwards        Pages: 84
    Friedreich Ataxia - Pipeline Review, H2 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Friedreich Ataxia - Pipeline Review, H2 2017, provides an overview of the Friedreich Ataxia (Central Nervous System) pipeline landscape. Friedreich's ataxia (FA) is a neuromuscular disease that mainly affects the nervous system and the heart. FA is a hereditary disease, caused by a defective gene that can be passed dow......
  • Opium (Opioid) Addiction - Pipeline Review, H2 2017
    Published: 12-Sep-2017        Price: US 2000 Onwards        Pages: 114
    Opium (Opioid) Addiction - Pipeline Review, H2 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Opium (Opioid) Addiction - Pipeline Review, H2 2017, provides an overview of the Opium (Opioid) Addiction (Central Nervous System) pipeline landscape. Opioid Addiction develops after using opioids regularly for a period of time. Prolonged use of opiates can lead to nerve damage within the brain that causes cells......
  • 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