Compassionate use of human recombinant insulin-like growth factor-1 therapy in Friedreich's ataxia

García Ron A, Rodriguez Mesa M. , Compassionate use of human recombinant insulin-like growth factor-1 therapy in Friedreich's ataxia del tratamiento como uso compasivo de mecasermina (factor de crecimiento insulínico recombinante humano tipo 1) en ataxia de Friedreich. Neurología. 2019. doi:10.1016/j.nrl.2017.11.004

As no curative treatment is currently available for FA, we believe that any drug potentially capable of halting or delaying disease progression without causing severe adverse effects merits further research. Further randomised controlled trials are needed to confirm the benefits obtained in our case, to try to determine the action mechanism of rhIGF-1, and to establish what can be expected during the long-term treatment of this and other neurodegenerative diseases.