Concurrent nanotherapeutics and regulatory updates for the management of amyotrophic lateral sclerosis: a focused review for orphan drug (Tofersen)
Integrating regulatory updates, clinical evidence, and innovative therapeutic strategies, the review uniquely contributes to the ALS literature by bridging current treatment realities with potential future therapies, aiming to inform researchers, clinicians, and policymakers on optimizing ALS management.
Authors
- Priya Pathak (ORCID: https://orcid.org/0000-0002-4150-0058)
- Shivang Shukla (ORCID: https://orcid.org/0009-0002-6395-6838)
- Kumar Pranav Narayan (ORCID: https://orcid.org/0000-0001-5198-2495)
- Abhiram Kumar (ORCID: https://orcid.org/0000-0002-0232-6317)
- Amit Rai (ORCID: https://orcid.org/0009-0009-9037-325X)
Institutions
- Jagannath University (IN)
- Birla Institute of Technology and Science - Hyderabad Campus (IN)
- Amity University (AE)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-11-21
- DOI
- https://doi.org/10.1186/s13023-025-04042-2
- Primary Topic
- Amyotrophic Lateral Sclerosis Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00