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Friedreich Ataxia - Pipeline Review, H2 2018

Friedreich Ataxia - Pipeline Review, H2 2018

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Friedreich Ataxia - Pipeline Review, H2 2018, provides an overview of the Friedreich Ataxi


Introduction
Global Markets Direct Report Coverage
Friedreich Ataxia - Overview
Friedreich Ataxia - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Friedreich Ataxia - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Friedreich Ataxia - Companies Involved in Therapeutics Development
Adverum Biotechnologies Inc
Agios Pharmaceuticals Inc
BioElectron Technology Corp
BioMarin Pharmaceutical Inc
Biovista Inc
Horizon Pharma Plc
Ixchel Pharma LLC
Pfizer Inc
PTC Therapeutics Inc
Reata Pharmaceuticals Inc
STATegics Inc
Takeda Pharmaceutical Co Ltd
Voyager Therapeutics Inc
Friedreich Ataxia - Drug Profiles
(sodium phenylbutyrate + tauroursodeoxycholic acid) - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ADVM-063 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AGIL-FA - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BMN-290 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BVA-202 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BVA-203 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Cell Therapy for Central Nervous System and Neurodegenerative Diseases - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CTI-1601 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
dextro epicatechin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Frataxin for Friedrich's Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
interferon gamma-1b - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
IXC-103 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JOT-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
omaveloxolone - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RT-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RT-002 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules for Friedreich Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate Frataxin for Friedreich Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
STSE-15 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
TAK-831 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
vatiquinone - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VYFXN-01 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Friedreich Ataxia - Dormant Projects
Friedreich Ataxia - Discontinued Products
Friedreich Ataxia - Product Development Milestones
Featured News & Press Releases
May 18, 2018: Voyager Therapeutics Announces Additional Data at the American Society of Gene and Cell Therapy 2018 Annual Meeting
Aug 22, 2017: Jupiter Orphan Therapeutics Receives Orphan Drug Designation for its Trans-Resveratrol Product JOTROL for Treatment of Friedreich's Ataxia
Aug 03, 2017: Chondrial Announces FDA Orphan Drug Designation for CTI-1601, a Novel Investigational Technology for the Treatment of Friedreich's Ataxia
Dec 08, 2016: Horizon Pharma Announces Top line Results from Phase 3 Study of ACTIMMUNE (interferon gamma-1b) in Friedreich's Ataxia
May 05, 2016: Horizon Pharma Completes Target Enrollment of 90 Patients for Phase 3 Trial of ACTIMMUNE(R) (interferon gamma-1b) for the Treatment of People With Friedreich Ataxia
Aug 05, 2015: Key Patent Granted for AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia
Jun 05, 2015: Horizon Pharma Initiates Phase 3 Trial of ACTIMMUNE for the Treatment of People With Friedreich's Ataxia
Apr 10, 2015: Horizon Pharma Receives FDA Fast Track Designation for ACTIMMUNE in the Treatment of Friedreich's Ataxia
Feb 13, 2015: Horizon Pharma Submits Investigational New Drug Application for ACTIMMUNE in the Treatment of Friedreich's Ataxia
Oct 09, 2014: Horizon Pharma Announces Presentation of ACTIMMUNE Phase 2 Data in Friedreich's Ataxia
Oct 03, 2014: Horizon Pharma Receives Orphan-Drug Designation for ACTIMMUNE (Interferon gamma-1b) in Friedreich's Ataxia
Mar 17, 2014: FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia
Feb 04, 2014: FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Friedreich's Ataxia
Nov 04, 2013: Edison Pharmaceuticals, FARA, and USF Announce Initiation of EPI-743 Phase 2 Friedreich's Ataxia Clinical Trial in Adults with Point Mutations
Jan 08, 2013: STATegics Announces a New Grant from Friedreich’s Ataxia Research Alliance
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Number of Products under Development for Friedreich Ataxia, H2 2018
Number of Products under Development by Companies, 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 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
Friedreich Ataxia - Pipeline by Adverum Biotechnologies Inc, H2 2018
Friedreich Ataxia - Pipeline by Agios Pharmaceuticals Inc, H2 2018
Friedreich Ataxia - Pipeline by BioElectron Technology Corp, H2 2018
Friedreich Ataxia - Pipeline by BioMarin Pharmaceutical Inc, H2 2018
Friedreich Ataxia - Pipeline by Biovista Inc, H2 2018
Friedreich Ataxia - Pipeline by Horizon Pharma Plc, H2 2018
Friedreich Ataxia - Pipeline by Ixchel Pharma LLC, H2 2018
Friedreich Ataxia - Pipeline by Pfizer Inc, H2 2018
Friedreich Ataxia - Pipeline by PTC Therapeutics Inc, H2 2018
Friedreich Ataxia - Pipeline by Reata Pharmaceuticals Inc, H2 2018
Friedreich Ataxia - Pipeline by STATegics Inc, H2 2018
Friedreich Ataxia - Pipeline by Takeda Pharmaceutical Co Ltd, H2 2018
Friedreich Ataxia - Pipeline by Voyager Therapeutics Inc, H2 2018
Friedreich Ataxia - Dormant Projects, H2 2018
Friedreich Ataxia - Discontinued Products, H2 2018
List of Figures
Number of Products under Development for Friedreich Ataxia, 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 Targets, H2 2018
Number of Products by Stage and Targets, H2 2018
Number of Products by Mechanism of Actions, H2 2018
Number of Products by Stage and 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

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