December 6, 2021
Pierre, suffering from limb girdle muscular dystrophy: “For me, a cure would mean no more losing”
Pierre is a 14-year-old middle-schooler. Four years ago he received the diagnosis on FKRP limb girdle muscular dystrophy. This progressive disease saps his strength little by little. At Genethon, Isabelle Richard’s team has worked for over 25 years on limb girdle muscular dystrophy. A first gene therapy treatment for Pierre’s disease could… (read more)
Spinal muscular atrophy: for Victoire’s mom, treatment was a magical moment
Suffering from spinal muscular atrophy, Victoire received a gene therapy treatment derived from technologies developed at Genethon. Her mom recounts the anguish of the diagnosis, the immense relief of the treatment and the little girl’s spectacular progress in the weeks following the injection.
(read more)June 28, 2021
Preliminary results of clinical trial for rare liver disease Crigler-Najjar syndrome presented at ESGCT congress
May 27, 2021
Cholesterol Metabolism: A Potential Therapeutic Target in Duchenne Muscular Dystrophy
David Israeli, his student Ai Vu Hong PhD, and Fatima Amor, all members of the Progressive Dystrophies laboratory directed by Isabelle Richard at Genethon, have published the results of research offering new perspectives on the treatment of Duchenne muscular dystrophy in the (read more)
May 10, 2021
Genethon’s teams present their progress at the ASGCT meeting
Limb girdle muscular dystrophies, rare diseases of the liver or production of vectors on a large scale: these are some of the topics presented by Genethon’s teams at the annual meeting of the American Society for Gene and Cell Therapy (ASGCT), to be held virtually from May 11 to 14.
(read more)April 23, 2021
Genethon announces First Patient dosed in Clinical Trial of Investigational Gene therapy GNT 0004 for Duchenne Muscular Dystrophy
February 28, 2021
Genethon, 30 years of pioneering research and innovation in treating rare diseases
February 10, 2021
Genethon and WhiteLab Genomics join forces to enhance gene therapy through artificial intelligence
February 3, 2021
Genethon is delighted about the launch of a gene therapy clinical trial for late-onset Pompe disease
December 2, 2020
Genethon gets the green light from the ANSM to start an innovative gene therapy trial for Duchenne muscular dystrophy
September 21, 2020
Genethon welcomes the submission to EMA of Marketing Authorisation Application for LUMEVOQ® Gene Therapy to Treat Vision Loss due to Leber Hereditary Optic Neuropathy (LHON)
GenSight Biologics, a french biopharma company, just submitted to EMAMarketing Autorisation Application for LUMEVOQ® Gene Therapy to Treat Vision Loss due to Leber Hereditary Optic Neuropathy (LHON), a rare, mitochondrial genetic disease, caused by mutation in the ND4 mitochondrial gene.
(read more)June 10, 2020
A Genethon team succeeds in inhibiting the immune response linked to AAV, opening up the possibility of treating more patients by gene therapy
January 30, 2020
Genethon welcomes the conclusive results of a gene therapy trial in Chronic Septic Granulomatosis, a rare disease of the immune system
January 9, 2020
Genethon strengthens its collaboration with Sarepta Therapeutics for the development of the gene therapy product GNT0004 in Duchenne muscular dystrophy
September 9, 2019
