High‐throughput screening identifies bazedoxifene as a potential therapeutic for dysferlin‐deficient limb girdle muscular dystrophy
Our drug screening identified saracatinib and bazedoxifene as potential treatments for LGMD R2, especially for patients with the L1341P mutation. The widespread protective effect of bazedoxifene reveals a new avenue toward genotype-independent treatment of LGMD R2 patients.
Authors
- Nathalie Bourg
- Anne Bigot (ORCID: https://orcid.org/0000-0003-0337-5425)
- Jérôme Polentes
- Manon Benabidès
- Johana Tournois (ORCID: https://orcid.org/0000-0003-0696-1307)
- Emilie Pellier
- Anthony Brureau
- Isabelle Richard (ORCID: https://orcid.org/0000-0002-6505-446X)
- Xavier Nissan (ORCID: https://orcid.org/0000-0003-3374-6274)
- Céline Bruge (ORCID: https://orcid.org/0000-0002-9505-5527)
- Noëlla Grossi
Institutions
- Inserm (FR)
- Université Paris-Saclay (FR)
- Sorbonne Université (FR)
- Institut de Myologie (FR)
- Genethon (France) (FR)
- Centre de Recherche en Myologie
- Université d'Évry Val-d'Essonne (FR)
Publication Details
- Journal
- British Journal of Pharmacology
- Published
- 2025-03-19
- DOI
- https://doi.org/10.1111/bph.70017
- Citations
- 2
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 1.61
Funders
- French Muscular Dystrophy Association
- European Commission