Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia

Mercado-Ayón E, Warren N, Halawani S, Rodden LN, Ngaba L, Dong YN, Chang JC, Fonck C, Mavilio F, Lynch DR and Lin H (2022). Front. Neurosci. 16:819569. doi: 10.3389/fnins.2022.819569 

Taken together, our findings show that frataxin knockdown leads to cerebellar degeneration in the FRDAkd mouse model, suggesting that frataxin helps maintain cerebellar structure and function.

The U.S. Food and Drug Administration granted orphan drug designation to elamipretide to treat Friedreich’s ataxia (FRDA).

 U.S. Food and Drug Administration, 03/22/2022

Generic Name:	elamipretide
Date Designated:	03/22/2022
Orphan Designation:	Treatment of Friedreich’s ataxia (FRDA)
Orphan Designation Status:	Designated
FDA Orphan Approval Status:	Not FDA Approved for Orphan Indication
Sponsor:	Stealth BioTherapeutics 275 Grove Street, Suite 3-107 Newton, Massachusetts 02466 United States The sponsor address listed is the last reported by the sponsor to OOPD.