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Atamyo Therapeutics obtains authorisation to launch a gene therapy clinical trial in limb-girdle muscular dystrophy linked to SGCG

Following several proofs of concept, Atamyo Therapeutics, a spin-off from Geenethon, is to start a new gene therapy trial in patients suffering from limb-girdle muscular dystrophy (LGMD) R5 linked to SGCG (gamma-sarcoglycanopathy). Its aim is to test the safety, pharmacodynamics and efficacy of ATA-200, a product resulting from the work of Isabelle Richard, a researcher … Continued
08 April 2024

Vianney, mentor of the Telethon, on 8 & 9 December

After Matt Pokora, Soprano and Kev Adams, it’s singer and composer Vianney’s turn to join forces with researchers, families and thousands of volunteers across France in the fight against rare diseases on 8 and 9 December 2023. The ambassador families for the next Telethon reflect the victories won thanks to you, against diseases that were once incurable, and … Continued
13 September 2023

2023, every gesture counts

Dear patients and their families,Dear donors,Dear partners, All the teams and collaborators of the Institute of Myology send you their best wishes for happiness and success and wish you excellent health. May this new year, full of promise, be synonymous with progress and success and enable us to accomplish the projects we cherish. We would … Continued
05 January 2023

SAPPHIRE trial starts at I-Motion

The SAPPHIRE trial is an international, randomised, double-blind, placebo-controlled phase III trial that will evaluate the effects of a dual treatment : apitegromab (an anti-myostatin developed by Scholar Rock) injected intravenously every 4 weeks for one year, in addition to treatment with nusinersen or risdiplam, in non-walking patients with SMA type II or III, 156 … Continued
30 November 2022

December 2 and 3: ready for the Telethon!

You live abroad and want to play a part in the Telethon? Join the Telethon for French people abroad and help us break records of mobilization around the world!  On December 2 and 3, 2022, the Telethon will rally together millions of French people to support research and enable the AFM-Telethon to fulfill its missions … Continued
08 November 2022
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