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A Phase 1a Double-Blind, Randomized, Placebo-Controlled, Single and Multiple Ascending Dose Study of the Safety, Pharmacokinetics, and Pharmacodynamics of ABX-002 in Healthy Adult Subjects

Part 1 Single Ascending Dose (SAD)- To evaluate the safety and tolerability of a single oral dose of prodrug, ABX-002, in healthy adult subjectsPart 2 Multiple Ascending Dose (MAD)-To evaluate the safety and tolerability of once daily oral doses of…

Quantifying disease progression in LBSL

Objective: The primary objective of this proposal is to develop new sensitive and quantitative outcome measures for clinical trials in LBSL. The secondary objective is to identify biomarkers to stratify patients based on disease progression rate.

Prospective, Retrospective, Multicenter, Observational Study of Disease Progression in Adults with Inherited Forms of Spastic Paraplegia

Study Objectives:• To characterize disease progression (clinical and rehabilitation) in adults diagnosed with AMN• To characterize the change in Quality of Life (QoL) parameters• To characterize the change in electrophysiological (EP) parameters• To…

Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

This study has been transitioned to CTIS with ID 2024-513904-33-00 check the CTIS register for the current data. • Monitor for long-term safety of the eli-cel administered in parent clinical studies• Monitor for long-term efficacy of eli-cel…