Categoria: Progetti finanziati

AavantiBio riceve il finanziamento di 107 milioni di dollari per l’avanzamento del suo programma di terapia genica per l’ Atassia di Friedreich

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GoFAR partners with AavantiBio, Inc. to advance gene therapy for Friedreich’s Ataxia.

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GoFAR reinforces partnership with Powell Gene Therapy Center at University of Florida (UF) to develop a gene therapy for Friedreich’s ataxia

International advocacy group GoFAR funds UF Health research for Friedreich’s ataxia gene therapy program

GoFAR rinforza la partnership con il Powell Gene Therapy Center dell’Università della Florida (UF) per sviluppare una terapia genica per l’atassia di Friedreich

UF and the Advocacy Group GoFAR Announce Gene Therapy program for Friedreich’s Ataxia.



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Early and longitudinal assessment of neurodegeneration in the brain and spinal cord in Friedreich’s ataxia (II year)

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Progetto: Early and longitudinal assessment of neurodegeneration in the brain and spinal cord in Friedreich’s ataxia (II year)

Principal Investigators: Pierre-Gilles Henry, Ph.D, Christophe Lenglet, Ph.D – Center for Magnetic Resonance Research, Department of Radiology,University of Minnesota Medical School, Minneapolis US

We propose to assess the sensitivity of candidate biomarkers forFriedreich’s ataxia in patients, using Magnetic Resonance Imaging (MRI) techniques.Sensitive early-stage biomarkers of the disease are currently lacking in Friedreich’s Ataxia (FRDA).Such biomarkers would constitute an invaluable tool for monitoring disease progression in prospective therapeutic trials.In this project, we propose to use both Magnetic Resonance Spectroscopy (MRS) and diffusionMagnetic Resonance Imaging (diffusion MRI). MRS allows non-invasive measurement of a“neurochemical profile” of 10-15 metabolites, and has the potential to reveal biochemical alterations in the nervous system before irreversible tissue damage. Diffusion MRI, in combination with advanced white matter fiber orientation mapping and tractography algorithms, provides unprecedented information about the integrity of the cerebral, cerebellar and spinal axonal wiring.Preliminary data obtained in our center in the past year shows significant differences in both MRS and diffusion MRI data between patients and controls. In this project based on our preliminary results, we propose to focus on a subset of the data already acquired and investigate year-to-year temporal changes in Friedreich’s ataxia.We hope to identify sensitive biomarkers of the disease in the central nervous system of patients with Friedreich’s ataxia at an early stage, and gain better insights into their temporal evolution. These aims could ultimately provide novel and unique tools for the quantitativeassessment of treatment efficacy in prospective therapeutic trials.

Finanziamento: 150,000 $ (1anno da 1 Settembre 2015) Co-finanziato da FARA e Ataxia UK