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PUBLICATIONS AND PRESENTATIONS INTENDED FOR MEDICAL PROFESSIONALS

Medical Publications - All Dates

Molecular Genetics and Metabolism - 07/16/2025

Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial

Molecular Genetics and Metabolism Reports - 06/17/2025

Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale

Molecular Genetics and Metabolism - 04/11/2025

Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C disease

Patterson et al - 02/12/2021

Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale

Mengel et al - 11/23/2020

Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study

Journal of Inherited Metabolic Disease - 08/21/2021

Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment

Pharmacoepidemiology & Drug Safety - 08/03/2017

Patterns of abuse and routes of administration for immediate-release hydrocodone combination products

Medical Presentations - All Dates

International Niemann-Pick Disease Alliance (INPDA) Meeting - 09/20/2025

Changing The Course of NPC: Long-term Evidence for Disease Modification in a Heterogenous Population

International Congress of Inborn Errors of Metabolism (ICIEM) 2025 - 09/04/2025

Safety and efficacy of arimoclomol in a pediatric substudy of Niemann-Pick disease type C patients aged 6 to <24 months at study enrollment

International Congress of Inborn Errors of Metabolism (ICIEM) 2025 - 09/04/2025

Arimoclomol upregulates expression of genes belonging to the coordinated lysosomal expression and regulation (CLEAR) network

International Congress of Inborn Errors of Metabolism (ICIEM) 2025 - 09/04/2025

Arimoclomol for the treatment of Niemann-Pick disease type C in a real-world setting: long-term outcomes from an expanded access program in the United States

International Congress of Inborn Errors of Metabolism (ICIEM) 2025 - 09/04/2025

Efficacy results across a 12-month double-blind randomized trial and an open-label extension phase of arimoclomol for treatment of Niemann-Pick disease type C in patients treated with miglustat

SERGG 2025 - 07/20/2025

ADMINISTRATION OF A DUAL-COATED SODIUM PHENYLBUTYRATE (OLPRUVA) SUSPENSION VIA GASTROSTOMY TUBE

SERGG 2025 - 07/20/2025

Arimoclomol for the Treatment of Niemann-Pick Disease Type C in a Real-World Setting: Long-Term Outcomes From an Expanded Access Program in the United States

SERGG 2025 - 07/20/2025

Long-Term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients With Niemann-Pick Disease Type C – Data From a 48-Month Open-Label Trial

SERGG 2025 - 07/20/2025

Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network

National Niemann Pick Disease Foundation (NNPDF) Conference - 07/11/2025

Long-Term Effectiveness and Safety Evaluation of Arimoclomol Treatment in Patients With Niemann-Pick Disease Type C – Data From the Pivotal Study and Open-Label Extension

National Niemann Pick Disease Foundation (NNPDF) Conference - 07/11/2025

Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network

National Niemann Pick Disease Foundation (NNPDF) Conference - 07/11/2025

Advances in Niemann-Pick Disease Type C Treatment: The Role of Arimoclomol

21st Annual WORLDSymposium - 02/07/2025

Efficacy Results from a 12-month Double-blind Randomized Trial of Arimoclomol for the Treatment of Niemann-Pick Disease Type C — Presenting a Rescored 4-domain NPC Clinical Severity Scale

21st Annual WORLDSymposium - 02/05/2025

Qualitative Assessment of the Validity and Standardization of the Swallow Domain in the 5-domain Niemann-Pick Disease Type C (NPC) Clinical Severity Scale (5DNPCCSS) And Analysis in an NPC Clinical Trial Data Set

21st Annual WORLDSymposium - 02/05/2025

Arimoclomol Safety Profile in the Treatment of Niemann-pick Disease Type C In a Real-world Setting: Long-term Safety Data from an Expanded Access Program in the United States

21st Annual WORLDSymposium - 02/05/2025

Long-term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients with Niemann-Pick Type C – Data From 48 Months Open Label Trial

21st Annual WORLDSymposium - 02/05/2025

Safety of Arimoclomol in a Pediatric Sub-Study of Niemann-pick Disease Type C Patients Aged 6 To <24 Months at Study Enrollment

21st Annual WORLDSymposium - 02/05/2025

Perseverance Is Key for Regulatory Success in Ultra-Rare Diseases – Key Learnings from Arimoclomol’s Regulatory Journey

21st Annual WORLDSymposium - 02/05/2025

Arimoclomol for the Treatment of Niemann-pick Disease Type C In a Real-world Setting: Long-term Data from an Expanded Access Program in the United States

21st Annual WORLDSymposium - 02/05/2025

Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network

21st Annual WORLDSymposium - 02/05/2025

Efficacy Results from A 12-month Double-blind Randomised Trial of Arimoclomol for Treatment of Niemann-Pick Disease Type C – Presenting A Rescored 4-domain NPC Clinical Severity Scale

The Child Neurology Society Annual Meeting - 11/11/2024

Qualitative Assessment of the Validity and Standardization of the Swallow Domain in the 5-Domain Niemann-Pick disease type C (NPC) Clinical Severity Scale (5DNPCCSS)

American Society of Human Genetics 2024 Annual Meeting - 11/8/2024

Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network

Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium - 09/06/2024

Modeling the pharmacokinetics of phenylbutyrate in fed and fasted states

Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium - 09/06/2024

Arimoclomol for the Treatment of NPC in a Real-World Setting: Long-Term Outcomes from an Expanded Access Program in the USA

Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium - 09/06/2024

Arimoclomol Safety Profile in the Treatment of NPC in a Real-World Setting: Long-Term Data From an Expanded Access Program in the USA

Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium - 09/06/2024

Long-term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients with Niemann Pick Type C – Data from 48 Months Open Label Trial

Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium - 09/06/2024

Efficacy Results from a 12-month Double-blind Randomized Trial of Arimoclomol for Treatment of Niemann Pick Disease Type C – Presenting an Improved 4-Domain NPC Clinical Severity Scale

Society for Inherited Metabolic Disorders (SIMD) - 04/15/2024

Arimoclomol in adults with NPC in a real-world setting: Long-term data from an expanded access program in the USA

52nd Child Neurology Society Annual Meeting - 10/06/2023

Evaluation of the Long-Term Effect of Arimoclomol in NPC – 48 Months Data from CT-ORZY-NPC-002

52nd Child Neurology Society Annual Meeting - 10/05/2023

Real World Data Collection in Niemann-Pick Disease Type C – Data from Expanded Access Program with Arimoclomol

The 19th Annual WORLDSymposium^TM - February 22 – 26, 2023

Association Between NPC Severity Score Domains and Corresponding Items of the Performance-based Scale for the Assessment and Rating of Ataxia (SARA)

The 19th Annual WORLDSymposium^TM - February 22 – 26, 2023

Evaluation of the Long-Term Effect of Arimoclomol in NPC

American Academy of Pain Medicine (AAPM) Annual Meeting - March 15-19, 2017

Pharmacokinetics and Abuse Potential of Benzhydrocodone, A Novel Prodrug of Hydrocodone, After Intranasal Administration in Recreational Drug Users

American Academy of Pain Medicine (AAPM) Annual Meeting - March 15-19, 2017

Oral pharmacokinetics of KP511, a prodrug of hydromorphone, relative to hydromorphone in human volunteers

2017 International Conference on Opioids (ICOO) - June 11-3, 2016

Pharmacokinetics and Abuse Potential of KP511, a Novel Prodrug of Hydromorphone, after Intranasal Administration in Recreation Drug Users

American Professional Society for ADHD and Related Disorders - January 12-14, 2018

Single- and Multiple-dose Pharmacokinetics of KP415, a Novel d-Methylphenidate Product Containing a Prodrug of d-Methylphenidate, in Healthy Volunteers

American Academy of Child & Adolescent Psychiatry - October 22-27, 2018

Dose-finding Study of Abuse-Related Effects of Intranasal d-Methylphenidate in Recreational Stimulant Abusers

American Academy of Child & Adolescent Psychiatry - October 22-27, 2018

Human Abuse Potential of Intravenous Serdexmethylphenidate (SDX), a Novel Prodrug of d-Methylphenidate, in Recreational Stimulant Abusers

American College of Neuropsychopharmacology - December 9-13, 2018

Assessment of the Intravenous Abuse Potential Of Serdexmethylphenidate (SDX), a Novel, Investigational Prodrug of D-Methylphenidate: Evidence from Nonclinical and Clinical Studies

The American Professional Society of ADHD and Related Disorders - January 18-20, 2019

Human Abuse Potential of Intranasal Serdexmethylphenidate (SDX), a Novel Prodrug of d-Methylphenidate, in Recreational Stimulant Abusers

The American Professional Society of ADHD and Related Disorders - January 18-20, 2019

Human Abuse Potential of Oral Serdexmethylphenidate (SDX), a Novel Prodrug of d-Methylphenidate, Compared to Focalin® XR and Phentermine in Recreational Stimulant Abusers

American Academy of Child and Adolescent Psychiatry - October 12-24, 2020

Patterns of Abuse of Prescription Stimulant Products from the National Addictions Vigilance Intervention Program (NAVIPPRO): 2010-2018

The American Professional Society of ADHD and Related Disorders (APSARD) 2022 Annual Conference - January 14-16, 2022

Dose Proportionality and Effects of Food on the Pharmacokinetics of Single-Entity Serdexmethylphenidate

The American Professional Society of ADHD and Related Disorders (APSARD) 2022 Annual Conference - January 14-16, 2022

Mass Balance and Metabolic Pathway Following Oral Administration of [14C]-Serdexmethylphenidate, a Novel Prodrug of d-Methylphenidate

Medical Publications - 2026

Medical Presentations - 2026

Medical Publications - 2025 to 2024

Molecular Genetics and Metabolism - 07/16/2025

Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial

Molecular Genetics and Metabolism Reports - 06/17/2025

Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale

Molecular Genetics and Metabolism - 04/11/2025

Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C disease

Medical Presentations - 2025 to 2024

International Niemann-Pick Disease Alliance (INPDA) Meeting - 09/20/2025

Changing The Course of NPC: Long-term Evidence for Disease Modification in a Heterogenous Population

International Congress of Inborn Errors of Metabolism (ICIEM) 2025 - 09/04/2025

Safety and efficacy of arimoclomol in a pediatric substudy of Niemann-Pick disease type C patients aged 6 to <24 months at study enrollment

International Congress of Inborn Errors of Metabolism (ICIEM) 2025 - 09/04/2025

Arimoclomol upregulates expression of genes belonging to the coordinated lysosomal expression and regulation (CLEAR) network

International Congress of Inborn Errors of Metabolism (ICIEM) 2025 - 09/04/2025

Arimoclomol for the treatment of Niemann-Pick disease type C in a real-world setting: long-term outcomes from an expanded access program in the United States

International Congress of Inborn Errors of Metabolism (ICIEM) 2025 - 09/04/2025

Efficacy results across a 12-month double-blind randomized trial and an open-label extension phase of arimoclomol for treatment of Niemann-Pick disease type C in patients treated with miglustat

SERGG 2025 - 07/20/2025

ADMINISTRATION OF A DUAL-COATED SODIUM PHENYLBUTYRATE (OLPRUVA) SUSPENSION VIA GASTROSTOMY TUBE

SERGG 2025 - 07/20/2025

Arimoclomol for the Treatment of Niemann-Pick Disease Type C in a Real-World Setting: Long-Term Outcomes From an Expanded Access Program in the United States

SERGG 2025 - 07/20/2025

Long-Term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients With Niemann-Pick Disease Type C – Data From a 48-Month Open-Label Trial

SERGG 2025 - 07/20/2025

Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network

National Niemann Pick Disease Foundation (NNPDF) Conference - 07/11/2025

Long-Term Effectiveness and Safety Evaluation of Arimoclomol Treatment in Patients With Niemann-Pick Disease Type C – Data From the Pivotal Study and Open-Label Extension

National Niemann Pick Disease Foundation (NNPDF) Conference - 07/11/2025

Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network

National Niemann Pick Disease Foundation (NNPDF) Conference - 07/11/2025

Advances in Niemann-Pick Disease Type C Treatment: The Role of Arimoclomol

21st Annual WORLDSymposium - 02/07/2025

Efficacy Results from a 12-month Double-blind Randomized Trial of Arimoclomol for the Treatment of Niemann-Pick Disease Type C — Presenting a Rescored 4-domain NPC Clinical Severity Scale

21st Annual WORLDSymposium - 02/05/2025

Qualitative Assessment of the Validity and Standardization of the Swallow Domain in the 5-domain Niemann-Pick Disease Type C (NPC) Clinical Severity Scale (5DNPCCSS) And Analysis in an NPC Clinical Trial Data Set

21st Annual WORLDSymposium - 02/05/2025

Arimoclomol Safety Profile in the Treatment of Niemann-pick Disease Type C In a Real-world Setting: Long-term Safety Data from an Expanded Access Program in the United States

21st Annual WORLDSymposium - 02/05/2025

Long-term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients with Niemann-Pick Type C – Data From 48 Months Open Label Trial

21st Annual WORLDSymposium - 02/05/2025

Safety of Arimoclomol in a Pediatric Sub-Study of Niemann-pick Disease Type C Patients Aged 6 To <24 Months at Study Enrollment

21st Annual WORLDSymposium - 02/05/2025

Perseverance Is Key for Regulatory Success in Ultra-Rare Diseases – Key Learnings from Arimoclomol’s Regulatory Journey

21st Annual WORLDSymposium - 02/05/2025

Arimoclomol for the Treatment of Niemann-pick Disease Type C In a Real-world Setting: Long-term Data from an Expanded Access Program in the United States

21st Annual WORLDSymposium - 02/05/2025

Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network

21st Annual WORLDSymposium - 02/05/2025

Efficacy Results from A 12-month Double-blind Randomised Trial of Arimoclomol for Treatment of Niemann-Pick Disease Type C – Presenting A Rescored 4-domain NPC Clinical Severity Scale

The Child Neurology Society Annual Meeting - 11/11/2024

Qualitative Assessment of the Validity and Standardization of the Swallow Domain in the 5-Domain Niemann-Pick disease type C (NPC) Clinical Severity Scale (5DNPCCSS)

American Society of Human Genetics 2024 Annual Meeting - 11/8/2024

Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network

Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium - 09/06/2024

Modeling the pharmacokinetics of phenylbutyrate in fed and fasted states

Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium - 09/06/2024

Arimoclomol for the Treatment of NPC in a Real-World Setting: Long-Term Outcomes from an Expanded Access Program in the USA

Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium - 09/06/2024

Arimoclomol Safety Profile in the Treatment of NPC in a Real-World Setting: Long-Term Data From an Expanded Access Program in the USA

Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium - 09/06/2024

Long-term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients with Niemann Pick Type C – Data from 48 Months Open Label Trial

Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium - 09/06/2024

Efficacy Results from a 12-month Double-blind Randomized Trial of Arimoclomol for Treatment of Niemann Pick Disease Type C – Presenting an Improved 4-Domain NPC Clinical Severity Scale

Society for Inherited Metabolic Disorders (SIMD) - 04/15/2024

Arimoclomol in adults with NPC in a real-world setting: Long-term data from an expanded access program in the USA

Medical Presentations - 2023 to 2022

52nd Child Neurology Society Annual Meeting - 10/06/2023

Evaluation of the Long-Term Effect of Arimoclomol in NPC – 48 Months Data from CT-ORZY-NPC-002

52nd Child Neurology Society Annual Meeting - 10/05/2023

Real World Data Collection in Niemann-Pick Disease Type C – Data from Expanded Access Program with Arimoclomol

The 19th Annual WORLDSymposium^TM - February 22 – 26, 2023

Association Between NPC Severity Score Domains and Corresponding Items of the Performance-based Scale for the Assessment and Rating of Ataxia (SARA)

The 19th Annual WORLDSymposium^TM - February 22 – 26, 2023

Evaluation of the Long-Term Effect of Arimoclomol in NPC

The American Professional Society of ADHD and Related Disorders (APSARD) 2022 Annual Conference - January 14-16, 2022

Dose Proportionality and Effects of Food on the Pharmacokinetics of Single-Entity Serdexmethylphenidate

The American Professional Society of ADHD and Related Disorders (APSARD) 2022 Annual Conference - January 14-16, 2022

Mass Balance and Metabolic Pathway Following Oral Administration of [14C]-Serdexmethylphenidate, a Novel Prodrug of d-Methylphenidate

Medical Publications - 2021 to 2020

Patterson et al - 02/12/2021

Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale

Mengel et al - 11/23/2020

Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study

Journal of Inherited Metabolic Disease - 08/21/2021

Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment

Medical Presentations - 2021 to 2020

American Academy of Child and Adolescent Psychiatry - October 12-24, 2020

Patterns of Abuse of Prescription Stimulant Products from the National Addictions Vigilance Intervention Program (NAVIPPRO): 2010-2018

Medical Publications - 2019 to 2018

Medical Presentations - 2019 to 2018

American Professional Society for ADHD and Related Disorders - January 12-14, 2018

Single- and Multiple-dose Pharmacokinetics of KP415, a Novel d-Methylphenidate Product Containing a Prodrug of d-Methylphenidate, in Healthy Volunteers

American Academy of Child & Adolescent Psychiatry - October 22-27, 2018

Dose-finding Study of Abuse-Related Effects of Intranasal d-Methylphenidate in Recreational Stimulant Abusers

American Academy of Child & Adolescent Psychiatry - October 22-27, 2018

Human Abuse Potential of Intravenous Serdexmethylphenidate (SDX), a Novel Prodrug of d-Methylphenidate, in Recreational Stimulant Abusers

American College of Neuropsychopharmacology - December 9-13, 2018

Assessment of the Intravenous Abuse Potential Of Serdexmethylphenidate (SDX), a Novel, Investigational Prodrug of D-Methylphenidate: Evidence from Nonclinical and Clinical Studies

The American Professional Society of ADHD and Related Disorders - January 18-20, 2019

Human Abuse Potential of Intranasal Serdexmethylphenidate (SDX), a Novel Prodrug of d-Methylphenidate, in Recreational Stimulant Abusers

The American Professional Society of ADHD and Related Disorders - January 18-20, 2019

Human Abuse Potential of Oral Serdexmethylphenidate (SDX), a Novel Prodrug of d-Methylphenidate, Compared to Focalin® XR and Phentermine in Recreational Stimulant Abusers

Medical Publications - 2017 to older

Pharmacoepidemiology & Drug Safety - 08/03/2017

Patterns of abuse and routes of administration for immediate-release hydrocodone combination products

Medical Presentations - 2017 to older

American Academy of Pain Medicine (AAPM) Annual Meeting - March 15-19, 2017

Pharmacokinetics and Abuse Potential of Benzhydrocodone, A Novel Prodrug of Hydrocodone, After Intranasal Administration in Recreational Drug Users

American Academy of Pain Medicine (AAPM) Annual Meeting - March 15-19, 2017

Oral pharmacokinetics of KP511, a prodrug of hydromorphone, relative to hydromorphone in human volunteers

2017 International Conference on Opioids (ICOO) - June 11-3, 2016