Duchenne muscular dystrophy: initial results of Genethon's gene therapy trial
The initial results of Genethon's gene therapy trial for Duchenne muscular dystrophy were presented on Tuesday 23 April at the Myology 2024 congress in Paris.
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The initial results of Genethon's gene therapy trial for Duchenne muscular dystrophy were presented on Tuesday 23 April at the Myology 2024 congress in Paris.
The AFM-Téléthon sends you its best wishes for the New Year and hopes that 2024 will be full of scientific breakthroughs, small and large joys, and shared victories!
The Telethon 2023 ended on a high of €80,671,222, which shows the confidence and exceptional mobilization of donors and volunteers for this year's event, rich in victories against disease and promises for all those waiting for the diagnosis and treatment that will change their lives. We must not give up.
The 8th edition of international congress dedicated to neuromuscular science, MYOLOGY 2024, will take place in Paris from 22 to 25 April 2024. Registration and call for abstract are now open : don't miss an opportunity to showcase your latest study to an international community of scientists and healthcare professionals. The deadline for abstract submission has been extended until November 30, 2023 - 11:59 pm.
The 30th of September is Limb-Girdle Muscular Dystrophy Awareness Day. At this occasion, the LGMD Interest Group of the AFM-Téléthon is planning an information day about these diseases in Evry.
"Advances in Charcot-Marie-Tooth (CMT) disease" is a new document, published by the French Muscular Dystrophy Association (AFM-Téléthon), which can be read and/or downloaded here.
The Téléthon 30-hour-long broadcast ended with donations totaling 78 051 091 euros. From Guebwiller to Cassis, Dijon, Lorient and Cap-Ferret, millions of people came together again for the Téléthon, this unique celebration with its thousands of events. A vital support for the families and researchers, “these saving lives maniacs” as Kev Adams called them. But while researchers’ victories multiply, all week-end long patients and their families reminded everyone of the urgency of finding new treatments.
The AFM-Telethon sends you its best wishes for the New Year and wishes you a year 2023 full of color and joy!
Read more about AFM-Telethon’s social missions, main activities, and key figures in this new edition of Cure through Innovation.
From September 12 to 17, 2022, AFM-Telethon will hold two international scientific congresses at the Nice Acropolis Convention Centre. You can submit your abstracts until April 30, 2022
It is a positive commitment, but France must set an example by perpetuating and amplifying a truly ambitious national policy.
On 16 December 2021, the UN adopted the first-ever UN Resolution on “Addressing the Challenges of Persons Living with a Rare Disease and their Families.” A huge recognition for families with rare diseases.
The 2021 Telethon ended with donations totaling 73 622 019 euros. An amount reflecting the generosity of the general public, the energy felt during thousands of Telethon events throughout France and abroad, and the strength of families’ fight and researchers’ determination. This unique combination is the cause for a true revolution in medicine that saves lives and opens up new prospects for treatment.
Genethon announced that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.