Sections
Title | Starting Page | Number of Pages |
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Table of Contents | 2 | 4 |
List of Tables | 5 | 1 |
List of Figures | 5 | 1 |
Introduction | 6 | 1 |
Global Markets Direct Report Coverage | 6 | 1 |
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Overview | 7 | 1 |
Therapeutics Development | 8 | 2 |
Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Overview | 8 | 1 |
Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Comparative Analysis | 9 | 1 |
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics under Development by Companies | 10 | 1 |
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics under Investigation by Universities/Institutes | 11 | 1 |
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Pipeline Products Glance | 12 | 3 |
Late Stage Products | 12 | 1 |
Clinical Stage Products | 13 | 1 |
Early Stage Products | 14 | 1 |
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Products under Development by Companies | 15 | 1 |
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Products under Investigation by Universities/Institutes | 16 | 1 |
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Companies Involved in Therapeutics Development | 17 | 5 |
CTD Holdings, Inc. | 17 | 1 |
La Jolla Pharmaceutical Company | 18 | 1 |
Merck &Co., Inc. | 19 | 1 |
Okklo Life Sciences BV | 20 | 1 |
Orphazyme ApS | 21 | 1 |
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics Assessment | 22 | 9 |
Assessment by Monotherapy Products | 22 | 1 |
Assessment by Target | 23 | 2 |
Assessment by Mechanism of Action | 25 | 2 |
Assessment by Route of Administration | 27 | 2 |
Assessment by Molecule Type | 29 | 2 |
Drug Profiles | 31 | 27 |
Antibodies for Metabolic Disorders Drug Profile | 31 | 1 |
arimoclomol Drug Profile | 32 | 2 |
Bryostatin-1 Drug Profile | 34 | 5 |
Delta-tocopherol Drug Profile | 39 | 1 |
HPGCD Drug Profile | 40 | 1 |
LJPC-0712 Drug Profile | 41 | 1 |
MLSA-1 Drug Profile | 42 | 1 |
OKL-1014 Drug Profile | 43 | 1 |
OR-0005 Drug Profile | 44 | 1 |
PERL-101 Drug Profile | 45 | 1 |
Small Molecule for Niemann-Pick Type C Disease Drug Profile | 46 | 1 |
Small Molecules for Niemann-Pick Type C Disease Drug Profile | 47 | 1 |
Small Molecules to Target Cholesterol for Niemann-Pick Type C Disease Drug Profile | 48 | 1 |
Trappsol Cyclo Drug Profile | 49 | 3 |
vorinostat Drug Profile | 52 | 4 |
VTS-270 Drug Profile | 56 | 2 |
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Dormant Projects | 58 | 1 |
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Product Development Milestones | 59 | 12 |
Featured News &Press Releases | 59 | 1 |
Sep 15, 2016: CTD Holdings Announces MHRA Approval of Clinical Trial Application for Treatment of NPC With Trappsol Cyclo | 59 | 1 |
Sep 12, 2016: CTD Holdings Files Clinical Trial Application With Swedish Medical Products Agency to Continue Advancing Its Drug Development Program | 60 | 1 |
Sep 06, 2016: CTD Holdings Announces FDA Acceptance of Investigational New Drug Application for Treatment of NPC With Trappsol Cyclo | 60 | 1 |
Aug 16, 2016: CTD Holdings Meets With Parent Advocate Groups and Physicians | 61 | 1 |
Aug 08, 2016: CTD Holdings Files Investigational New Drug Application With US FDA to Further Advance Its Drug Development Program | 62 | 1 |
Aug 01, 2016: CTD Holdings Files Clinical Trial Application with U.K. Regulatory Agency to Advance Its Drug Development Program | 63 | 1 |
Jun 28, 2016: CTD Holdings Announces Joint Presentation With UCSF Benioff Children Hospital Oakland on Patient Experience in Compassionate Treatment Program | 64 | 1 |
May 03, 2016: CTD Holdings to Present "Management of Niemann-Pick Type C With Intravenous Hydroxypropyl- b -Cyclodextrin" at Ara Parseghian Medical Research Foundation Annual Conference | 64 | 1 |
Feb 29, 2016: CTD Holdings Clinical Program to Move Forward in the U.S. | 65 | 1 |
Feb 18, 2016: Parent Advocates Join CTD Holdings Family and Physicians Listening Circle on NPC Treatment | 66 | 1 |
Dec 14, 2015: CTD Holdings' Clinical Trial Design Receives Positive Responses From U.K.'s Medicines and Healthcare Products Regulatory Agency | 66 | 1 |
Sep 16, 2015: N. Scott Fine Named CEO of CTD Holdings | 67 | 1 |
Jun 24, 2015: Neurotrope to Conduct Study on Niemann-Pick Type C, a Devastating Rare Pediatric Disease | 68 | 1 |
Jun 12, 2015: Neurotrope to Present Findings of In-vitro Studies of Bryostatin's Effects on Niemann Pick Type C1 Cells at the Annual "Michael, Marcia &Christa Parseghian Scientific Conference" for Niemann-Pick Type C | 69 | 1 |
Jan 27, 2015: FDA Accepts CTD Holdings Drug Master File for Second-Generation Orphan Drug | 70 | 1 |
Appendix | 71 | 2 |
Methodology | 71 | 1 |
Coverage | 71 | 1 |
Secondary Research | 71 | 1 |
Primary Research | 71 | 1 |
Expert Panel Validation | 71 | 1 |
Contact Us | 71 | 1 |
Disclaimer | 72 | 1 |