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

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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

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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

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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

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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

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SERGG 2025

- 07/20/2025

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

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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

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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

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SERGG 2025

- 07/20/2025

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

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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

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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

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National Niemann Pick Disease Foundation (NNPDF) Conference

- 07/11/2025

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

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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

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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

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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

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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

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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

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21st Annual WORLDSymposium

- 02/05/2025

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

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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

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21st Annual WORLDSymposium

- 02/05/2025

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

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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

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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)

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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)

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The 19th Annual WORLDSymposium^TM

- February 22 – 26, 2023

Evaluation of the Long-Term Effect of Arimoclomol in NPC

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Academy of Managed Care Pharmacy (AMCP)

- March 29 – April 1, 2022

Serdexmethylphenidate/d-Methylphenidate Capsules for Children With ADHD: Effects on SKAMP-C Evaluated Over 13 Hours in a Randomized, Double-blind, Placebo-controlled Laboratory Classroom Study

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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

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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

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Children and Adults with Attention-Deficit/Hyperactiviity Disorder

- November 4–6, 2021

Serdexmethylphenidate/d-Methylphenidate Capsules for Children With ADHD: Effects on SKAMP-C Evaluated Over 13 Hours in a Randomized, Double-blind, Placebo-controlled Laboratory Classroom Study

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Psych Congress

- October 29–November 1, 2021

Steady-State Pharmacokinetics and Relative Bioavailability of Serdexmethylphenidate/d-Methylphenidate, a Treatment for Attention Deficit Hyperactivity Disorder Containing a Novel Prodrug of d-Methylphenidate

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American Academy of Child & Adolescent Psychiatry

- October 18-30 2021

Single-dose Pharmacokinetics of Serdexmethylphenidate/d-Methylphenidate Capsules in Children and Adolescents With ADHD and Healthy Adults: An Evaluation of Age and Body Weight

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American Psychiatric Nurses Association

- October 13-16, 2021

Serdexmethylphenidate/d-Methylphenidate Capsules for Children With ADHD: Effects on SKAMP-C and WREMB-R as Evaluated in a Randomized, Double-blind, Placebo-controlled Laboratory Classroom Study

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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

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American Academy of Child and Adolescent Psychiatry

- October 14-19, 2019

Effect of Food and Alternative Dosing Methods on the Pharmacokinetics of KP415, an Investigational ADHD Product Containing the Prodrug, Serdexmethylphenidate (SDX)

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The American Professional Society of ADHD and Related Disorders

- January 18-20, 2019

Single-dose Pharmacokinetics of KP415, an Investigational Product Containing the Prodrug Serdexmethylphenidate (SDX), in Children and Adolescents with ADHD

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The American Professional Society of ADHD and Related Disorders

- January 18-20, 2019

Dose-Proportionality and Steady-State Pharmacokinetics of KP415, an Investigational ADHD Product Containing Serdexmethylphenidate (SDX), a Novel Prodrug of d-Methylphenidate

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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

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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

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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

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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

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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

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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

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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

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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

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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

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