Breaking news
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
The INCEPTUS study confirms the frequency of liver damage in X-linked myotubular myopathy
The international INCEPTUS study, a natural history study prior to the ASPIRO gene therapy trial to which I-Motion contributed, involved 34 children under 4 years of age with X-linked myotubular myopathy on respiratory support. During a median follow-up of 13 months (from 5 months to more than 2.5 years), there were three deaths (from inhalation … Continued
11 October 2022
