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      Global Cystic Fibrosis Strategic Business Report 2017 - Research and Markets

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      News provided by

      Research and Markets

      20 Mar, 2017, 18:45 GMT

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      DUBLIN, Mar 20, 2017 /PRNewswire/ --

      Research and Markets has announced the addition of the "Cystic Fibrosis - Global Strategic Business Report" report to their offering.

      This report analyzes the worldwide markets for Cystic Fibrosis in US$ Million. The report provides separate comprehensive analytics for the US, Canada, Europe, and Rest of World. Annual estimates and forecasts are provided for the period 2014 through 2022. Market data and analytics are derived from primary and secondary research.

      The report profiles 56 companies including many key and niche players such as

      • Advanced Inhalation Therapies (AIT) Ltd (Israel)
      • Alaxia SAS (France)
      • AlgiPharma AS (Norway)
      • Anthera Pharmaceuticals, Inc. (USA)
      • Arch BioPartners (Canada)
      • Celtaxsys, Inc. (USA)
      • Concert Pharmaceuticals, Inc. (USA)
      • Corbus Pharmaceuticals Holdings, Inc.(USA)
      • CURx Pharmaceuticals (USA)
      • Nivalis Therapeutics, Inc. (USA)
      • NovaBiotics Ltd (UK)
      • Parion Sciences, Inc. (USA)
      • Pharmaxis Ltd (Australia)
      • PTC Therapeutics, Inc. (USA)
      • Pulmatrix, Inc. (USA)
      • AbbVie Inc. (USA)
      • Galapagos NV (Belgium)
      • Genentech, Inc. (USA)
      • Gilead Sciences, Inc. (USA)
      • Novartis AG (Switzerland)
      • Vertex Pharmaceuticals (USA)

      Key Topics Covered:

      1. MARKET OVERVIEW
      Cystic Fibrosis
      A Prelude
      CF Disease Prevalence: A Brief Note
      Treatment Modalities for Cystic Fibrosis
      CFTR Modulators: Key Drug Category
      Competitive Scenario

      2. RESEARCH IN CYSTIC FIBROSIS SPACE
      Areas of Research & Development
      Gene Therapy
      A Step Towards Cure
      Research Findings & Innovations
      Updated CF Diagnosis Guidelines to Aid in Diagnosis and Personalized Treatment
      Pipeline Drugs
      An Overview
      Select Pipeline Drugs for Cystic Fibrosis Treatment by Development Stage, 2016
      Description of Select Pipeline Drugs
      Ataluren
      VX-661 + Ivacaftor
      Riociguat
      QBW251
      CTP-656
      Cavosonstat (N91115)
      VX-440
      QR-010
      pGM169/GL67A
      Acebilustat
      Resunab
      Inhaled Mannitol
      VX-371
      Inhaled Levoflaxacin (Quinsair)
      AeroVanc (Vancomycin Inhalation Powder)

      3. DISEASE OVERVIEW
      Cystic Fibrosis
      A Hereditary Disease
      Mutation in CFTR Gene
      A Major Cause of CF
      ?F508
      The Most Common Mutation that Affects CFTR Protein
      Hereditary Risk of the Disease
      Pathophysiology of the Disease
      Symptoms of Cystic Fibrosis
      Common Symptoms in Early Childhood
      Common Symptoms in Grownups or Adults:
      Complications Associated with Cystic Fibrosis
      Sinus and Lung Illness
      Pancreatic, Gastrointestinal, and Liver Disease
      Endocrine Disorders
      Infertility
      Diagnosis of Cystic Fibrosis
      Newborn Test
      Sweat Test
      The IRT Test
      Carrier Test
      Antenatal Test
      Other Common Tests
      Prenatal Diagnosis of Cystic Fibrosis
      Tests to Diagnose CF-Related Complications
      Cystic Fibrosis in Children

      4. CYSTIC FIBROSIS TREATMENT: AN OVERVIEW
      Existing Treatment Options for Cystic Fibrosis
      A Quick Glance at Common Drugs for Cystic Fibrosis Management
      Analysis of Drugs Indicated for Management of Cystic Fibrosis
      Antibiotic Therapy for Lung Infections
      Select Antibiotics Approved for CF Treatment
      TOBI (Tobramycin Inhalation Solution)
      Other Common Cystic Fibrosis Infections and Recommended Antibiotics
      Cayston (Aztreonam for Inhalation Solution)
      Combination Antibiotic Therapy
      Mucolytics
      Pulmozyme
      The Effective Mucolytic Agent
      Pancreatic Enzyme Replacement Therapy (PERT) for EPI
      Leading PE Drugs Worldwide for Treating Pancreatic Insufficiency Associated with Cystic Fibrosis
      CTFR Modulators
      Bronchodilators
      Additional Treatment for Patients with CF
      Surgical and Other Techniques
      Lung Transplantation
      Recommended for Rare Cases of CF
      Feeding Tube
      For Extra Nutrition
      Bowel Surgery
      To Remove any Blockage in Bowel

      5. CLINICAL STUDIES
      Vertex Pharmaceuticals Announces Positive Phase 3 Study Results of ORKAMBI
      ProQR Therapeutics Announces Positive Proof-of-Concept Study for QR-010
      Concert Pharmaceuticals Presents Positive Food Effect Results for CTP-656
      Vertex Pharmaceuticals Initiates Phase 2 Studies for VX-440 & VX-152
      Vertex Pharmaceuticals Provides Update on Phase 3 Study of VX
      661 with ivacaftor
      Protalix BioTherapeutics Announces Start of Phase II Trial of AIR DNase (PRX-110)
      Nivalis Therapeutics Announces Completion of Enrollment for Phase 2 Study of N91115
      Alaxia SAS Announces Agreement and Grant from CFFT for ALX-009 Studies
      Polyphor Completes Successful Phase I Study of Inhaled POL6014
      AlgiPharma Receives Grant from CFFT for Phase 2 Trials of OligoG
      Health Canada Grants Clearance to ProMetic for its PBI-4050
      Cystic Fibrosis Canada Finances Laurent Pharmaceuticals' LAU- 7b Phase 2 Trial
      Galapagos NV Begins Phase 2 Study of GLPG1837
      Pharmaxis Announces Positive Phase II Trial Results of Bronchitol®
      PTC Therapeutics Completes Enrollment for second Phase 3 clinical trial of Translarna
      Anthera Pharmaceuticals Begins Phase 3 Study of Oral Liprotamase
      Parion Sciences Announces Expanded Enrollment Criteria for CLEAN-CF Trial
      Laurent Announces Positive Phase 1b Trial Results for LAU-7b
      Celtaxsys Announces Positive Phase 1 Clinical Trial Data of CTX-4430
      Corbus Pharmaceuticals Receives USFDA Clearance to Initiate Phase 2 Study of Resunab
      Savara Pharmaceuticals Unveils Positive Phase 2 Trial Results of AeroVanc
      KaloBios Reports Top-line Data from Phase 2 Study of KB001-A
      Galapagos NV Nominates GLPG2222 for Combination Therapy with GLPG1837
      Cystic Fibrosis Trust and NovaBiotics Partner for the Phase IIa Study of Lynovex
      PTC Therapeutics Initiates Phase 3 Trial of Translarna/ataluren
      Kamada to Start Clinical Study of Alpha-1 Antitrypsin (AAT) for Cystic Fibrosis

      6. PRODUCT LAUNCHES/APPROVALS
      EC Grants Orphan Drug Designation to Corbus Pharmaceuticals' Resunab
      FDA Approves Vertex's ORKAMBI® for Extended Age Group
      Pharmaxis Announces Approval of Bronchitol® in Russia
      FDA Grants Orphan Drug Status to Pulmatrix's CF Drug PUR1900
      ProQR Therapeutics Announces FDA Fast Track Designation for QR-010
      Alcresta Therapeutics Launches Relizorb Cartridge
      EC Grants Orphan Medicinal Product Designation to Arch Biopartners' AB569
      TGV-Inhalonix Receives Orphan Drug Designation for its Mul-1867
      RespirTech Launches Development of Remote Monitoring System for CF
      Vertex Pharmaceuticals Receives TGA Approval of ORKAMBI®
      AlgiPharma's OligoG Receives Orphan Drug Designation by FDA
      Proteostasis Therapeutics Receives FDA Fast Track designation for PTI-428
      Proteostasis Therapeutics Expands CF Drug Pipeline
      Vertex Pharmaceuticals Receives Two EU Approvals for KALYDECO®
      USPTO Issues Patent to OrPro Therapeutics' Theradux
      FDA Approves Vertex's ORKAMBI
      Advanced Inhalation Therapies Receives Orphan Medicinal Product Designation for AIT-CF from EMA
      FDA Approves New Age Group Indication for Vertex Pharmaceuticals' KALYDECO
      Sandoz Launches Tobramycin Inhalation Solution in the US
      FDA Approves sNDA for KALYDECO
      EC Grants Orphan Designation to Macrolide CF Drug by Synovo GmbH

      7. RECENT INDUSTRY ACTIVITY
      Pharmaxis Enters into Research Collaboration with Woolcock Institute
      Horizon Pharma Acquires Raptor Pharmaceutical
      AstraZeneca to Sell Drug Commercialization Rights to Pfizer
      Vertex Pharmaceuticals and Moderna Therapeutics Enter into Research Collaboration
      Editas Medicine Enters into Agreement with CFFT for CRISPR/ Cas9-based CF Drugs
      Galapagos and AbbVie Expand Agreement for CF Portfolio
      Raptor Pharmaceutical Acquires Quinsair from Tripex Pharmaceuticals
      Galapagos NV Enters into Licensing Agreement with the HUB Foundation
      Zambon SpA Acquires Nigaard Pharma
      ManRos and Cyclacel Enter into Licensing Agreement for Seliciclib
      Vertex and Parion Sciences Collaborate to Develop Investigational CF Treatment
      Ruthigen and Pulmatrix Enter into Merger Agreement
      N30 Pharmaceuticals Changes Name to Nivalis Therapeutics
      Anthera Pharmaceuticals Acquires Sollpura/liprotamase from Eli Lilly
      OPKO Health Acquires Inspiromatic dry powder inhaler from Inspiro Medical
      CURx Pharmaceuticals Enters into Global License Agreement with Gilead Sciences

      8. SELECT CLINICAL-STAGE PLAYERS
      Advanced Inhalation Therapies (AIT) Ltd (Israel)
      Alaxia SAS (France)
      AlgiPharma AS (Norway)
      Anthera Pharmaceuticals, Inc. (USA)
      Arch BioPartners (Canada)
      Celtaxsys, Inc. (USA)
      Concert Pharmaceuticals, Inc. (USA)
      Corbus Pharmaceuticals Holdings, Inc.(USA)
      CURx Pharmaceuticals (USA)
      Nivalis Therapeutics, Inc. (USA)
      NovaBiotics Ltd (UK)
      Parion Sciences, Inc. (USA)
      Pharmaxis Ltd (Australia)
      PTC Therapeutics, Inc. (USA)
      Pulmatrix, Inc. (USA)

      9. FOCUS ON SELECT GLOBAL PLAYERS
      AbbVie Inc. (USA)
      Galapagos NV (Belgium)
      Genentech, Inc. (USA)
      Gilead Sciences, Inc. (USA)
      Novartis AG (Switzerland)
      Vertex Pharmaceuticals (USA)

      10. GLOBAL MARKET PERSPECTIVE

      Total Companies Profiled: 56 (including Divisions/Subsidiaries 58)

      - The United States (30)
      - Canada (4)
      - Europe (20)
      - France (2)
      - Germany (2)
      - The United Kingdom (3)
      - Italy (2)
      - Rest of Europe (11)
      - Asia-Pacific (Excluding Japan) (1)
      - Middle East (3)

      For more information about this report visit http://www.researchandmarkets.com/research/v7p7vg/cystic_fibrosis

      Media Contact:

      Research and Markets
      Laura Wood, Senior Manager
      press@researchandmarkets.com

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