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SMARD1: a gene therapy clinical trial opened at Nationwide Children's Hospital in Columbus (USA)

Aggiornamento: 8 gen 2022

On 10 December 2021, the announcement of the opening of a clinical study of gene therapy in patients with mutations in the IGHMBP2 gene: SMARD1 / CMT2S was published.

The study is conducted at Nationwide Children's Hospital in Columbus in Ohio (United States) and expects to recruit approximately 10 patients. According to the study protocol, there is a single intrathecal administration of gene therapy, directly into the cerebrospinal fluid. This study represents the transfer to a clinical study on patients of the pre-clinical research carried out for several years in the laboratories of our "Centro Dino Ferrari" of the University of Milan - Ospedale Maggiore Policlinico by Dr. Nizzardo, together with Dr. Govoni and Prof. Corti, in collaboration with Dr. Kathrin Meyer of the Columbus research center. All this is possible thanks to the great support of SMASHSMARD USA and SMASHSMARD DE, the pre-clinical work of Dr. Cox of the Jackson laboratory, of Dr. Burghes and Dr. Arnold of Ohio State University. Thanks in particular to Alcyone Therapeutics for having significantly contributed to the pre-clinical studies and to the clinical trial that has now begun. More information is available at this link: To find out more, write to:

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