/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-06-17 17:01:342025-06-17 17:01:34Efficacy 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
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-04-17 07:30:582025-04-17 07:37:40Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C disease
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-06 15:42:412025-02-06 15:42:41Efficacy 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
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 11:37:542025-02-03 11:52:43Qualitative 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
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 11:36:262025-02-03 11:52:51Arimoclomol 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
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 11:33:002025-02-03 11:52:55Long-term Efficacy and Safety Evaluation of Arimoclomol Treatment in Patients with Niemann-Pick Type C – Data From 48 Months Open Label Trial
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 11:00:242025-02-03 12:02:48Safety of Arimoclomol in a Pediatric Sub-Study of Niemann-pick Disease Type C Patients Aged 6 To <24 Months at Study Enrollment
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 10:57:532025-02-03 12:02:53Perseverance Is Key for Regulatory Success in Ultra-Rare Diseases – Key Learnings from Arimoclomol’s Regulatory Journey
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 10:54:142025-02-03 12:03:00Arimoclomol 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
/wp-content/uploads/logo.svg00Jeff Lorber/wp-content/uploads/logo.svgJeff Lorber2025-02-03 10:51:412025-02-03 12:03:07Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network
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