Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Acid Sphingomyelise Deficiency (Niemann-Pick Disease) Type C - Overview
Acid Sphingomyelise Deficiency (Niemann-Pick Disease) Type C - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Acid Sphingomyelise Deficiency (Niemann-Pick Disease) Type C - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Acid Sphingomyelise Deficiency (Niemann-Pick Disease) Type C - Companies Involved in Therapeutics Development
Acid Sphingomyelise Deficiency (Niemann-Pick Disease) Type C - Drug Profiles
Acid Sphingomyelise Deficiency (Niemann-Pick Disease) Type C - Dormant Projects
Acid Sphingomyelise Deficiency (Niemann-Pick Disease) Type C - Product Development Milestones
Featured News & Press Releases
Jul 13, 2022: Cyclo Therapeutics to present at the World Orphan Drug Congress USA 2022
Jun 24, 2022: Cyclo Therapeutics to present at the 2022 NPC Patient and Family Conference hosted by the Australian NPC Disease Foundation
May 09, 2022: Cyclo Therapeutics to participate in the virtual investor Niemann-Pick Disease Type C spotlight event
Mar 24, 2022: Azafaros announces FDA Grant of Orphan Drug Desigtion for AZ-3102 in the treatment of Niemann-Pick Disease
Feb 14, 2022: Azafaros presents positive clinical and preclinical data supporting development of lead compound AZ-3102 in lysosomal storage disorders at the 18th Annual WORLDSymposium conference
Feb 09, 2022: Cyclo therapeutics selected to present overview of pivotal phase 3 study for lead candidate, Trappsol Cyclo, at WORLDSymposium 2022
Feb 03, 2022: Cyclo Therapeutics announces formation of global steering committee comprised of leading experts to Advise on the global phase 3 clinical development program for Trappsol Cyclo in Niemann-Pick Disease Type C
Oct 18, 2021: Cyclo Therapeutics announces abstract accepted for poster presentation at the 14th Intertiol Congress of Inborn Errors of Metabolism (ICIEM)
Oct 06, 2021: Cyclo Therapeutics to participate in the 2021 Virtual Cyclodextrin Conference Hosted by CycloLab
Jul 30, 2021: Cyclo Therapeutics announces new positive safety and efficacy data from ongoing phase 1 open-label extension study of Trappsol Cyclo for the treatment of Niemann-Pick disease type C1
Jul 27, 2021: Cyclo Therapeutics commences commercial-scale manufacturing for Trappsol Cyclo
Jun 21, 2021: Cyclo Therapeutics to present at the 2021 NPC patient and family conference hosted by the Australian NPC Disease Foundation
Jun 18, 2021: Cyclo Therapeutics starts enrolment in Phase III NPC1 treatment trial
Apr 27, 2021: Cyclo Therapeutics announces design of pivotal phase 3 study evaluating Trappsol Cyclo in Niemann-Pick Type C1
Mar 25, 2021: Cyclo Therapeutics meets primary efficacy endpoint in phase 1/2 trial from intravenous Trappsol Cyclo in rare disease Niemann-Pick Type C1 (NPC1)
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures

List of Figures
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Number of Products by Top 10 Targets, 2022
Number of Products by Stage and Top 10 Targets, 2022
Number of Products by Top 10 Mechanism of Actions, 2022
Number of Products by Stage and Top 10 Mechanism of Actions, 2022
Number of Products by Routes of Administration, 2022
Number of Products by Stage and Routes of Administration, 2022
Number of Products by Molecule Types, 2022
Number of Products by Stage and Top 10 Molecule Types, 2022

List of Tables

List of Tables
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Products under Development by Universities/Institutes, 2022
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects, 2022