New gene therapy success in a rare disease

French teams from CIC Biothérapie (AP-HP/Inserm), from pediatric hematology department of Necker Hospital for Children (AP-HP), led by Marina Cavazzana, Salima Hacein Bey Albina and Alain Fischer and from Genethon led by Anne Galy (Genethon/Inserm UMR-S951), and English teams from UCL Institute of Child Health and Great Ormond Street Hospital in London led by Adrian Thrasher and Bobby Gaspar demonstrated the efficacy of gene therapy treatment for Wiskott-Aldrich Syndrome (WAS).

Six children that were treated and followed for at least 9 months
had their immune system restored and clinical condition improved. This work, which
was published today in the Journal of the American Medical Association (JAMA), was
carried out with support from the AFM-Telethon.
Syndrome (WAS).

21 Apr 2015

09 Apr 2015

Telethon 2014 : 92 920 108 €

An exceptional result, a fundraiser success, audience and mobilization

16 Jan 2015

Sarepta Therapeutics Announces First Patient Dosed in Duchenne Muscular Dystrophy Patients

Sarepta Therapeutics Inc. a developer of RNA-‐based therapeutics, announced that it has initiated dosing of SRP-‐4053 in its first human trial, a Phase I/II study in Duchenne muscula rdystrophy (DMD).

All the news