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Willeke Van Roon-Mom

11PUBLICATIONS
47CO-AUTHORS
Neurology and neuromuscular diseasesGene and molecular therapyMedical molecular engineering of nucleic acids and proteinsAutonomic nervous system
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Journal

Publications (11)

Sort by Publication Date:
|Apr 14, 2025
Treatability of the KMT2-Associated Neurodevelopmental Disorders Using Antisense Oligonucleotide-Based Treatments.

Bianca Zardetto, Willeke van Roon-Mom, Annemieke Aartsma-Rus

|Apr 14, 2025
Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments.

Bianca Zardetto, Marlen C Lauffer, Willeke van Roon-Mom

|Aug 15, 2023
From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of <i>SOD1</i> Mutated Amyotrophic Lateral Sclerosis.

Willeke van Roon-Mom, Chantal Ferguson, Annemieke Aartsma-Rus

|Jul 27, 2023
Machine learning in Huntington's disease: exploring the Enroll-HD dataset for prognosis and driving capability prediction.

Jasper Ouwerkerk, Stephanie Feleus, Kasper F van der Zwaan

|Jun 06, 2023
Spinocerebellar Ataxia Type 1 Characteristics in Patient-Derived Fibroblast and iPSC-Derived Neuronal Cultures.

Ronald A M Buijsen, Michel Hu, Maria Sáez-González

|May 27, 2023
Therapeutic Strategies for Spinocerebellar Ataxia Type 1.

Laurie M C Kerkhof, Bart P C van de Warrenburg, Willeke M C van Roon-Mom

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Frequent Collaborators

6 joint publications

Annemieke Aartsma-Rus

4 joint publications

Ronald A M Buijsen

3 joint publications

Marlen Lauffer

2 joint publications

Bart P C van de Warrenburg

2 joint publications

Bianca Zardetto

2 joint publications

Alejandro Garanto

2 joint publications

Eleni Mina

2 joint publications

Virginia Arechavala-Gomeza

2 joint publications

Aurelie T Goyenvalle

1 joint publications

Laurie M C Kerkhof

Frequent Collaborators

6 joint publications

Annemieke Aartsma-Rus

4 joint publications

Ronald A M Buijsen

3 joint publications

Marlen Lauffer

2 joint publications

Bart P C van de Warrenburg

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