Sections
| Title | Starting Page | Number of Pages |
|---|
| Table of Contents | 2 | 6 |
| List of Tables | 6 | 1 |
| List of Figures | 7 | 1 |
| Introduction | 8 | 1 |
| Global Markets Direct Report Coverage | 8 | 1 |
| Friedreich Ataxia Overview | 9 | 1 |
| Therapeutics Development | 10 | 2 |
| Pipeline Products for Friedreich Ataxia Overview | 10 | 1 |
| Pipeline Products for Friedreich Ataxia Comparative Analysis | 11 | 1 |
| Friedreich Ataxia Therapeutics under Development by Companies | 12 | 2 |
| Friedreich Ataxia Therapeutics under Investigation by Universities/Institutes | 14 | 1 |
| Friedreich Ataxia Pipeline Products Glance | 15 | 3 |
| Late Stage Products | 15 | 1 |
| Clinical Stage Products | 16 | 1 |
| Early Stage Products | 17 | 1 |
| Friedreich Ataxia Products under Development by Companies | 18 | 1 |
| Friedreich Ataxia Products under Investigation by Universities/Institutes | 19 | 1 |
| Friedreich Ataxia Companies Involved in Therapeutics Development | 20 | 15 |
| Adverum Biotechnologies, Inc. | 20 | 1 |
| Almirall, S.A. | 21 | 1 |
| BioMarin Pharmaceutical Inc. | 22 | 1 |
| Biovista Inc. | 23 | 1 |
| Catabasis Pharmaceuticals, Inc. | 24 | 1 |
| Edison Pharmaceuticals, Inc. | 25 | 1 |
| Horizon Pharma Plc | 26 | 1 |
| Ixchel Pharma, LLC | 27 | 1 |
| ProQR Therapeutics N.V. | 28 | 1 |
| RaNA Therapeutics, Inc. | 29 | 1 |
| Reata Pharmaceuticals, Inc. | 30 | 1 |
| Retrotope, Inc | 31 | 1 |
| Shire Plc | 32 | 1 |
| STATegics, Inc. | 33 | 1 |
| Voyager Therapeutics, Inc. | 34 | 1 |
| Friedreich Ataxia Therapeutics Assessment | 35 | 9 |
| Assessment by Monotherapy Products | 35 | 1 |
| Assessment by Target | 36 | 2 |
| Assessment by Mechanism of Action | 38 | 2 |
| Assessment by Route of Administration | 40 | 2 |
| Assessment by Molecule Type | 42 | 2 |
| Drug Profiles | 44 | 38 |
| AGIL-FA Drug Profile | 44 | 1 |
| ANN-003 Drug Profile | 45 | 2 |
| BVA-202 Drug Profile | 47 | 1 |
| BVA-203 Drug Profile | 48 | 1 |
| CAT-4001 Drug Profile | 49 | 1 |
| dimethyl fumarate Drug Profile | 50 | 1 |
| Gene Therapy to Activate Frataxin for Friedreich s Ataxia Drug Profile | 51 | 1 |
| Gene Therapy to Activate Frataxin for Friedreich s Ataxia Drug Profile | 52 | 1 |
| Gene Therapy to Activate Frataxin for Friedrich's Ataxia Drug Profile | 53 | 1 |
| interferon gamma-1b Drug Profile | 54 | 4 |
| interferon gamma-1b Drug Profile | 58 | 1 |
| IXC-101 Drug Profile | 59 | 1 |
| JOT-101 Drug Profile | 60 | 1 |
| Oligonucleotide to Activate Frataxin for Friedreich's Ataxia Drug Profile | 61 | 1 |
| omaveloxolone Drug Profile | 62 | 3 |
| QRX-604 Drug Profile | 65 | 1 |
| Recombinant Protein for Friedreich Ataxia Drug Profile | 66 | 1 |
| RG-3250 Drug Profile | 67 | 1 |
| RT-001 Drug Profile | 68 | 2 |
| RT-002 Drug Profile | 70 | 1 |
| SHP-622 Drug Profile | 71 | 2 |
| STSE-15 Drug Profile | 73 | 2 |
| TAT-FXN Drug Profile | 75 | 1 |
| vatiquinone Drug Profile | 76 | 5 |
| VYFXN-01 Drug Profile | 81 | 1 |
| Friedreich Ataxia Dormant Projects | 82 | 1 |
| Friedreich Ataxia Discontinued Products | 83 | 1 |
| Friedreich Ataxia Product Development Milestones | 84 | 10 |
| Featured News &Press Releases | 84 | 1 |
| Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich s Ataxia | 84 | 1 |
| 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 | 84 | 1 |
| Jan 19, 2016: FARA Announces Catabasis Pharmaceuticals as the Recipient of the Kyle Bryant Translational Research Award to Evaluate CAT-4001 as a Potential Therapy for Friedreich s Ataxia | 85 | 1 |
| Aug 05, 2015: Key Patent Granted For AAVLife s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich s Ataxia | 86 | 1 |
| Jun 05, 2015: Horizon Pharma Initiates Phase 3 Trial of ACTIMMUNE for the Treatment of People With Friedreich's Ataxia | 87 | 1 |
| Apr 10, 2015: Horizon Pharma Receives FDA Fast Track Designation for ACTIMMUNE in the Treatment of Friedreich's Ataxia | 87 | 1 |
| Mar 24, 2015: Catabasis Pharmaceuticals to Present at International Ataxia Research Conference | 88 | 1 |
| Feb 13, 2015: Horizon Pharma Submits Investigational New Drug Application for ACTIMMUNE in the Treatment of Friedreich's Ataxia | 88 | 1 |
| Jan 29, 2015: Reata Enrolls First Patient in the MOXIe Study, a Phase 2/3 Study Examining RTA 408 in Friedreich's Ataxia Patients | 89 | 1 |
| Oct 09, 2014: Horizon Pharma Announces Presentation of ACTIMMUNE Phase 2 Data in Friedreich's Ataxia | 89 | 1 |
| Oct 03, 2014: Horizon Pharma Receives Orphan-Drug Designation for ACTIMMUNE (Interferon gamma-1b) in Friedreich's Ataxia | 90 | 1 |
| Sep 30, 2014: Reata Announces the Initiation of Phase 2 Studies Examining RTA 408 for the Treatment of Friedreich's Ataxia and Mitochondrial Myopathies | 90 | 1 |
| Mar 17, 2014: FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia | 91 | 1 |
| Feb 04, 2014: FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Friedreich's Ataxia | 92 | 1 |
| 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 | 92 | 2 |
| Appendix | 94 | 2 |
| Methodology | 94 | 1 |
| Coverage | 94 | 1 |
| Secondary Research | 94 | 1 |
| Primary Research | 94 | 1 |
| Expert Panel Validation | 94 | 1 |
| Contact Us | 94 | 1 |
| Disclaimer | 95 | 1 |
