Long-term (30 Months) Outcomes of N-acetyl-L-leucine in Adults and Children with Niemann-Pick Disease Type C
Authors: Tatiana Bremova-Ertl, Jorgji Kerthi, Bethany Zanrucha, Janelle Raymond, Taylor Fields, Ian Billington, Michael Strupp, Marc C. Patterson
Published: European Academy of Neurology 2026 Annual Meeting
Date: June 27-30, 2026
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Results from the Global Phase 3 Clinical Trial (IB1001-303) Evaluating Levacetylleucine in Ataxia-Telangiectasia
Authors: Kyriakos Martakis, Marc C. Patterson, Michael Strupp, Bethany Zanrucha, Janelle Raymond, Jorgji Kerthi, Taylor Fields, Ian Billington, Tatiana Bremova-Ertl
Published: European Academy of Neurology 2026 Annual Meeting
Date: June 27-30, 2026
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Measuring Functional Abilities and Assistance Level in Niemann-Pick disease type C, GM2 gangliosidoses, and Ataxia
Authors: Bethany Zanrucha, Marc C. Patterson, Jorgji Kerthi, Janelle Raymond, Megan Grosso, Jake Caines, Susan Rounds
Published: European Academy of Neurology 2026 Annual Meeting
Date: June 27-30, 2026
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Results from the Global Phase 3 Trial (IB1001-303) Evaluating Levacetylleucine in Children and Adults with Ataxia-Telangiectasia
Authors: Marc C. Patterson, Tatiana Bremova-Ertl, Michael Strupp, Jorgji Kerthi, Janelle Raymond, Bethany Zanrucha, Asante Hatcher, Taylor Fields, Ian Billington, Kyriakos Martakis
Published: American Academy of Neurology 2025 Annual Meeting
Date: April 18-22, 2026
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Long-Term 30-Month Findings of N-acetyl-L-leucine for Niemann-Pick Disease Type C
Authors: Michael Strupp, Marc C. Patterson, Bethany Zanrucha, Janelle Raymond, Asante Hatcher, Jorgji Kerthi, Taylor Fields, Ian Billington, Tatiana Bremova-Ertl
Published: American Academy of Neurology 2025 Annual Meeting
Date: April 18-22, 2026
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Expanded Access Program of Levacetylleucine in Ataxia-Telangiectasia
Authors: Marc C. Patterson, Jorgji Kerthi, Megan Grosso, Bethany Zanrucha, Janelle Raymond, Asante Hatcher, Taylor Fields
Published: 6th Pan American Parkinson’s Disease and Movement Disorders Congress
Date: February 14, 2026
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Long-Term Findings of N-Acetyl-L-Leucine for Niemann-Pick Disease Type C
Authors: Marc C. Patterson, Bethany Zanrucha, Janelle Raymond, Asante Hatcher, Jorgji Kerthi, Taylor Fields, Ian Billington, Michael Strupp, Tatiana Bremova-Ertl
Published: 22nd Annual WorldSymposium 2026
Date: February 6, 2026
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Subdomain Analysis of the 5-Domain Niemann-Pick Disease Type C Clinical Severity Scale Evaluating the Long-Term Effects of N-Acetyl-L-Leucine in Niemann-Pick Disease Type C
Authors: Marc C. Patterson, Jorgji Kerthi, Bethany Zanrucha, Janelle Raymond, Asante Hatcher, Taylor Fields, Ian Billington, Michael Strupp, Tatiana Bremova-Ertl
Published: 22nd Annual WorldSymposium 2026
Date: February 5, 2026
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SARA Subgroup Analysis Based on Disease Duration in a Phase 2 Trial Evaluating N-Acetyl-L-Leucine in GM2 Gangliosidoses
Authors: Marc C. Patterson, Janelle Raymond, Jorgji Kerthi, Bethany Zanrucha, Asante Hatcher, Taylor Fields, Ian Billington, Michael Strupp, Tatiana Bremova-Ertl, Kyriakos Martakis
Published: 22nd Annual WorldSymposium 2026
Date: February 5, 2026
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Subgroup Evaluation of Adults and Children with Niemann-Pick Disease Type C in the Phase 3 (IB1001-301) Extension Phase Assessing N-Acetyl-L-Leucine
Authors: Marc C. Patterson, Jorgji Kerthi, Bethany Zanrucha, Janelle Raymond, Asante Hatcher, Taylor Fields, Ian Billington, Michael Strupp, Tatiana Bremova-Ertl
Published: 22nd Annual WorldSymposium 2026
Date: February 3, 2026
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The Functional Independence Measure for Children (WeeFIM®) in Niemann-Pick Disease Type C Receiving Levacetylleucine
Authors: Marc C. Patterson, Megan Grosso, Jake Caines, Susan Rounds
Published: 2nd Annual WorldSymposium 2026
Date: February 3, 2026
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N-Acetyl-L-Leucine for Ataxia-Telangiectasia: A Multinational, Double-Blind, Randomized, Placebo-Controlled, Crossover Study
Authors: Marc C. Patterson, Asante Hatcher, Janelle Raymond, Bethany Zanrucha, Taylor Fields, Michael Strupp, Tatiana Bremova-Ertl, Kyriakos Martakis
Published: Child Neurology Society 2025 Annual Meeting
Date: October 8-11, 2025
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Long-Term 24-Month Findings of N-acetyl-L-leucine for Niemann-Pick Disease Type C
Authors: Marc C. Patterson, Bethany Zanrucha, Asante Hatcher, Janelle Raymond, Taylor Fields, Michael Strupp, Tatiana Bremova-Ertl, Kyriakos Martakis
Published: International Congress of Inborn Errors of Metabolism 2025
Date: September 2-6, 2025
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Long-Term 52-Week Findings on N-Acetyl-L-Leucine for GM2 Gangliosidoses
Authors: Marc C. Patterson, Bethany Zanrucha, Asante Hatcher, Janelle Raymond, Taylor Fields, Michael Strupp, Kyriakos Martakis
Published: International Congress of Inborn Errors of Metabolism 2025
Date: September 2-6, 2025
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SCAFI Subtest and SARA Eight-Item Subgroup Analysis of N-Acetyl-L-Leucine in Phase II, Randomized, Rater-Blinded Crossover Trial for GM2 Gangliosidoses
Authors: Marc C. Patterson, Asante Hatcher, Janelle Raymond, Bethany Zanrucha, Taylor Fields, Ian Billington, Tatiana Bremova-Ertl, Kyriakos Martakis, Michael Strupp
Published: International Congress of Inborn Errors of Metabolism 2025
Date: September 2-6, 2025
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Long-Term 52-Week Findings on N-Acetyl-L-Leucine for GM2 Gangliosidoses (Tay-Sachs and Sandhoff Disease)
Authors: Bethany Zanrucha, Michael Strupp, Marc C. Patterson, Janelle Raymond, Asante Hatcher, Jorgji Kerthi, Taylor Fields, Tatiana Bremova-Ertl, Kyriakos Martakis
Published: World Conference in Pediatrics, Neonatology, and Infectious Diseases 2025 Annual Meeting
Date: July 14-15, 2025
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Long-Term 24-Month Findings of N-acetyl-L-leucine for Niemann-Pick Disease Type C
Authors: Michael Strupp, Marc C. Patterson, Janelle Raymond, Bethany Zanrucha, Asante Hatcher, Taylor Fields, Tatiana Bremova-Ertl, Kyriakos Martakis
Published: European Academy of Neurology 2025 Annual Meeting
Date: June 21-24, 2025
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SARA Eight-Item Subgroup Analysis of N-Acetyl-L-Leucine for Niemann-Pick Disease Type C
Authors: Marc C. Patterson, Bethany Zanrucha, Janelle Raymond, Asante Hatcher, Tatiana Bremova-Ertl, Michael Strupp, Ian Billington, Taylor Fields
Published: American Academy of Neurology 2025 Annual Meeting
Date: April 5-9, 2025
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Subgroup and Subdomain Analysis of Patients in a Phase III, Randomized, Placebo-Controlled Crossover Trial with Levacetylleucine (IB1001) for Niemann-Pick Disease Type C With Long Term Follow-Up
Authors: Marc C. Patterson, Bethany Zanrucha, Janelle Raymond, Asante Hatcher, Tatiana Bremova-Ertl, Michael Strupp, Ian Billington, Taylor Fields
Published: American Academy of Neurology 2025 Annual Meeting
Date: April 5-9, 2025
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Subgroup Analysis by Phenotype in a Phase III, Randomized, Placebo-Controlled Crossover Trial with Levacetylleucine (IB1001) for Niemann-Pick Disease Type C
Authors: Asante Hatcher, Bethany Zanrucha, Janelle Raymond, Michael Strupp, Tatiana Bremova-Ertl, Ian Billington, Taylor Fields
Published: American College of Medical Genetics and Genomics 2025 Annual Meeting
Date: March 18-22, 2025
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