IRDiRC announces publication of a new manuscript, developed as part of the International Rare Disease Research Consortium (IRDiRC) N-of-1 Task Force: “Applying the IRDiRC N-of-1 Therapy Task Force Eligibility Criteria for Individualised Therapies – Use Case: Duchenne Muscular Dystrophy“.
Nucleic acid-based therapies, including antisense oligonucleotides (ASOs), are opening new doors for the treatment of rare diseases. With over 30 individuals currently receiving N-of-1 ASO therapies, there is increasing interest in expanding these personalised approaches. However, the complexity of these treatments calls for a clear and ethical framework.
This publication tests the eligibility roadmap developed by the IRDiRC Task Force by applying it to a real-world use case: Duchenne muscular dystrophy (DMD).
Key Findings:
- ASOs offer meaningful opportunities for individualised treatment in rare diseases.
- Not all diseases or patients qualify under current eligibility criteria.
- DMD, despite significant research interest, does not yet fully meet the necessary criteria for individualised therapy development.
The study highlights both the promise and the challenges in bringing N-of-1 therapies from concept to clinic, emphasizing the need for careful evaluation of medical need, scientific feasibility, and ethical considerations.
Read the full article here: https://www.nmd-journal.com/article/S0960-8966(25)00948-4/fulltext