Annemieke Aartsma‐Rus

机构：Leiden University Medical Center, Duchenne Parent Project · ORCID：0000-0003-1565-654X

发表论文 429 篇 · 总被引 20278 次 · h-index 67

代表论文

The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing (2025 · Nucleic Acids Research · 被引 16)
Gene therapy approval for Duchenne muscular dystrophy: a European perspective (2025 · The Lancet · 被引 7)
Tailored antisense oligonucleotides for ultrarare CNS diseases: An experience-based best practice framework for individual patient evaluation (2025 · Molecular Therapy — Nucleic Acids · 被引 6)
Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: A retrospective cohort study (2025 · Genetics in Medicine · 被引 3)
Applying the international rare disease research consortium (IRDiRC) N-of-1 therapy task force eligibility criteria for individualised therapies use case: Duchenne muscular dystrophy (2025 · Neuromuscular Disorders · 被引 2)
Design and In Vitro Evaluation of a Novel Antisense Oligonucleotide for Treatment of BK Virus Infection (2025 · Nucleic Acid Therapeutics · 被引 2)