Drug Candidate BIO101 for Spinal Muscular Atrophy as Monotherapy or Combined With the Antisense Oligonucleotide ASO‐10‐27
We showed that BIO101 constitutes an efficient SMN-independent therapy to improve muscle performance in SMA, which could open new therapeutic avenues for patients in combination with SMN-based therapies, or as monotherapy for less severe forms.
Authors
- Frédéric Charbonnier (ORCID: https://orcid.org/0000-0003-4341-3246)
- René Lafont (ORCID: https://orcid.org/0000-0002-8044-540X)
- Steve Cottin
- Olivier Biondi (ORCID: https://orcid.org/0000-0003-4302-6035)
- Parvin Nazari Hashemi
- Mirella El Khoury (ORCID: https://orcid.org/0000-0001-6223-2526)
- Laure Weill (ORCID: https://orcid.org/0000-0003-3579-6582)
- Mathilde Latil (ORCID: https://orcid.org/0000-0002-8424-017X)
- Cynthia Bézier
- Christine Balducci (ORCID: https://orcid.org/0000-0001-9541-2902)
- Delphine Sapaly (ORCID: https://orcid.org/0000-0003-1112-2495)
- Pierre J. Dilda (ORCID: https://orcid.org/0000-0001-9916-8513)
- Stanislas Veillet (ORCID: https://orcid.org/0000-0003-2772-6015)
- Zoé Clerc
Institutions
- Délégation Paris 5 (FR)
- Centre National de la Recherche Scientifique (FR)
- Inserm (FR)
- Université Paris Cité (FR)
- Sorbonne Université (FR)
- Sorbonne Paris Cité (FR)
- Institut de Biologie Paris-Seine (FR)
- Biophytis (France) (FR)
- Université d'Évry Val-d'Essonne (FR)
Publication Details
- Journal
- Journal of Cachexia Sarcopenia and Muscle
- Published
- 2025-10-01
- DOI
- https://doi.org/10.1002/jcsm.70104
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00