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Pompe Disease ' Pipeline Review, H2 2012

Pompe Disease ' Pipeline Review, H2 2012 Summary Global Markets Direct's, 'Pompe Disease - Pipeline Review, H2 2012', provides an overview of the indication's therapeutic pipeline. This report provides information on the therapeutic development for Pompe Disease, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Pompe Disease. Pompe Disease - Pipeline Review, Half Year is built using data and information sourced from Global Markets Direct's proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team.Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease. Scope - A snapshot of the global therapeutic scenario for Pompe Disease.- A review of the Pompe Disease products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.- Coverage of products based on various stages of development ranging from discovery till registration stages.- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.- Coverage of the Pompe Disease pipeline on the basis of route of administration and molecule type.- Key discontinued pipeline projects.- Latest news and deals relating to the products. Reasons to buy - Identify and understand important and diverse types of therapeutics under development for Pompe Disease.- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.- Plan mergers and acquisitions effectively by identifying players of the most promising pipeline.- Devise corrective measures for pipeline projects by understanding Pompe Disease 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.- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline.
Published on: Mar 4, 2016
Source: www.slideshare.net


Transcripts - Pompe Disease ' Pipeline Review, H2 2012

  • 1. Find Industry reports, Company profilesReportLinker and Market Statistics >> Get this Report Now by email!Pompe Disease Pipeline Review, H2 2012Published on December 2012 Report SummaryPompe Disease Pipeline Review, H2 2012SummaryGlobal Markets Directs, Pompe Disease - Pipeline Review, H2 2012, provides an overview of the indications therapeutic pipeline.This report provides information on the therapeutic development for Pompe Disease, complete with latest updates, and specialfeatures on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for PompeDisease. Pompe Disease - Pipeline Review, Half Year is built using data and information sourced from Global Markets Directsproprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases fromcompany/university sites and industry-specific third party sources, put together by Global Markets Directs team.Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicateddisease.Scope- A snapshot of the global therapeutic scenario for Pompe Disease.- A review of the Pompe Disease products under development by companies and universities/research institutes based on informationderived from company and industry-specific sources.- Coverage of products based on various stages of development ranging from discovery till registration stages.- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.- Coverage of the Pompe Disease pipeline on the basis of route of administration and molecule type.- Key discontinued pipeline projects.- Latest news and deals relating to the products.Reasons to buy- Identify and understand important and diverse types of therapeutics under development for Pompe Disease.- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitiveadvantage.- Plan mergers and acquisitions effectively by identifying players of the most promising pipeline.- Devise corrective measures for pipeline projects by understanding Pompe Disease pipeline depth and focus of Indicationtherapeutics.- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects toenhance and expand business potential and scope.- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline.Pompe Disease Pipeline Review, H2 2012 (From Slideshare) Page 1/6
  • 2. Find Industry reports, Company profilesReportLinker and Market Statistics >> Get this Report Now by email! Table of ContentTable of ContentsTable of Contents 2List of Tables 4List of Figures 4Introduction 5Global Markets Direct Report Coverage 5Pompe Disease Overview 6Therapeutics Development 7An Overview of Pipeline Products for Pompe Disease 7Pompe Disease Therapeutics under Development by Companies 9Pompe Disease Therapeutics under Investigation by Universities/Institutes 10Mid Clinical Stage Products 11Comparative Analysis 11Pre-Clinical Stage Products 12Comparative Analysis 12Pompe Disease Therapeutics Products under Development by Companies 13Pompe Disease Therapeutics Products under Investigation by Universities/Institutes 14Companies Involved in Pompe Disease Therapeutics Development 15Genzyme Corporation 15Kaiser Permanente 16BioMarin Pharmaceutical Inc. 17Amicus Therapeutics, Inc. 18Pompe Disease Therapeutics Assessment 19Assessment by Monotherapy Products 19Assessment by Combination Products 20Assessment by Route of Administration 21Assessment by Molecule Type 23Drug Profiles 25duvoglustat hydrochloride - Drug Profile 25Product Description 25Mechanism of Action 25R&D Progress 25BMN-701 - Drug Profile 27Product Description 27Mechanism of Action 27R&D Progress 27rAAV1-CMV-GAA - Drug Profile 29Product Description 29Mechanism of Action 29R&D Progress 29Neo-rhGAA - Drug Profile 30Product Description 30Mechanism of Action 30R&D Progress 30Pompe Disease Pipeline Review, H2 2012 (From Slideshare) Page 2/6
  • 3. Find Industry reports, Company profilesReportLinker and Market Statistics >> Get this Report Now by email!AT-2220 + Alglucosidase Alfa (ERT) - Drug Profile 31Product Description 31Mechanism of Action 31R&D Progress 31BMN-103 - Drug Profile 32Product Description 32Mechanism of Action 32R&D Progress 32bortezomib - Drug Profile 33Product Description 33Mechanism of Action 33R&D Progress 33Pompe Disease Therapeutics Drug Profile Updates 34Pompe Disease Therapeutics - Dormant Products 36Pompe Disease Product Development Milestones 37Featured News & Press Releases 37Oct 11, 2012: Amicus Therapeutics Announces Positive Pompe Program Updates 37Jun 26, 2012: Amicus Announces Positive Preliminary Results From Phase II Chaperone-Enzyme Replacement TherapyCo-Administration Study For Pompe Disease 39Jan 21, 2010: Genzyme Announces Six-Month FDA Review For Lumizyme BLA 41Appendix 42Methodology 42Coverage 42Secondary Research 42Primary Research 42Expert Panel Validation 42Contact Us 43Disclaimer 43List of TablesNumber of Products Under Development for Pompe Disease, H2 2012 7Products under Development for Pompe Disease Comparative Analysis, H2 2012 8Number of Products under Development by Companies, H2 2012 9Number of Products under Investigation by Universities/Institutes, H2 2012 10Comparative Analysis by Mid Clinical Stage Development, H2 2012 11Comparative Analysis by Pre-Clinical Stage Development, H2 2012 12Products under Development by Companies, H2 2012 13Products under Investigation by Universities/Institutes, H2 2012 14Genzyme Corporation, H2 2012 15Kaiser Permanente, H2 2012 16BioMarin Pharmaceutical Inc., H2 2012 17Amicus Therapeutics, Inc., H2 2012 18Assessment by Monotherapy Products, H2 2012 19Assessment by Combination Products, H2 2012 20Assessment by Stage and Route of Administration, H2 2012 22Pompe Disease Pipeline Review, H2 2012 (From Slideshare) Page 3/6
  • 4. Find Industry reports, Company profilesReportLinker and Market Statistics >> Get this Report Now by email!Assessment by Stage and Molecule Type, H2 2012 24Pompe Disease Therapeutics Drug Profile Updates 34Pompe Disease Therapeutics Dormant Products 36List of FiguresNumber of Products under Development for Pompe Disease, H2 2012 7Products under Development for Pompe Disease Comparative Analysis, H2 2012 8Products under Development by Companies, H2 2012 9Products under Investigation by Universities/Institutes, H2 2012 10Mid Clinical Stage Products, H2 2012 11Pre-Clinical Stage Products, H2 2012 12Assessment by Monotherapy Products, H2 2012 19Assessment by Combination Products, H2 2012 20Assessment by Route of Administration, H2 2012 21Assessment by Stage and Route of Administration, H2 2012 22Assessment by Molecule Type, H2 2012 23Assessment by Stage and Molecule Type, H2 2012 24Pompe Disease Pipeline Review, H2 2012 (From Slideshare) Page 4/6
  • 5. Find Industry reports, Company profilesReportLinker and Market Statistics >> Get this Report Now by email! Fax Order Form To place an order via fax simply print this form, fill in the information below and fax the completed form to: Europe, Middle East and Africa : + 33 4 37 37 15 56 Asia, Oceania and America : + 1 (805) 617 17 93 If you have any questions please visit http://www.reportlinker.com/notify/contact Order Information Please verify that the product information is correct and select the format(s) you require. Pompe Disease Pipeline Review, H2 2012 Product Formats Please select the product formats and the quantity you require. 1 User License--USD 2 000.00 Quantity: _____ Site License--USD 4 000.00 Quantity: _____ Corporate License--USD 6 000.00 Quantity: _____ Contact Information Please enter all the information below in BLOCK CAPITALS Title: Mr Mrs Dr Miss Ms Prof First Name: _____________________________ Last Name: __________________________________ Email Address: __________________________________________________________________________ Job Title: __________________________________________________________________________ Organization: __________________________________________________________________________ Address: __________________________________________________________________________ City: __________________________________________________________________________ Postal / Zip Code: __________________________________________________________________________ Country: __________________________________________________________________________ Phone Number: __________________________________________________________________________ Fax Number: __________________________________________________________________________Pompe Disease Pipeline Review, H2 2012 (From Slideshare) Page 5/6
  • 6. Find Industry reports, Company profilesReportLinker and Market Statistics >> Get this Report Now by email! Payment Information Please indicate the payment method, you would like to use by selecting the appropriate box. Payment by credit card Card Number: ______________________________________________ Expiry Date __________ / _________ CVV Number _____________________ Card Type (ex: Visa, Amex…) _________________________________ Payment by wire transfer Crédit Mutuel RIB : 10278 07314 00020257701 89 BIC : CMCIFR2A IBAN : FR76 1027 8073 1400 0202 5770 189 Payment by check UBIQUICK SAS 16 rue Grenette – 69002 LYON, FRANCE Customer signature:   Please note that by ordering from Reportlinker you are agreeing to our Terms and Conditions at http://www.reportlinker.com/index/terms Please fax this form to: Europe, Middle East and Africa : + 33 4 37 37 15 56 Asia, Oceania and America : + 1 (805) 617 17 93Pompe Disease Pipeline Review, H2 2012 (From Slideshare) Page 6/6

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