da Admin | Mag 19, 2021 | Clinical
https://www.pharmastar.it Sarepta Therapeutics reported positive initial data from a phase I trial on SRP-9001 gene therapy, for patients with Duchenne muscular dystrophy (DMD). The ENDEAVOR study enrolled 20 DMD patients with ages between four and seven, who received...
da Admin | Mag 11, 2021 | Clinical
https://www.nejm.org A recent article published in “the New England Journal of Medicine” reported the results of a study on a gene therapy for the treatment of adenosine deaminase (ADA) deficiency (ADA-SCID). This type of deficiency is part of the severe...
da Admin | Mag 6, 2021 | Clinical
https://www.nature.com Preliminary results of the first pilot study on gene therapy for Fabry disease have been published in the “Nature Communication”. Fabry disease is a hereditary disease caused by a mutation in the GLA gene, which codes for the alpha-galactosidase...
da Admin | Apr 30, 2021 | Clinical
https://aadcnews.com A viral vector-delivered gene therapy can restore the activity of the AADC enzyme in people with AADC deficiency. This is the main results of an open-label Phase 1/2 trial that tested a gene therapy called AAV2-hAADC in eight young patients with...
da Admin | Apr 14, 2021 | Clinical
https://www.bionews.org.uk A clinical trial for a new gene therapy approach to treat sickle cell disease has been approved to proceed by the US Food and Drug Administration. Patients with sickle cell disease have a mutation in the β -haemoglobin gene, causing them to...
da Admin | Apr 12, 2021 | Clinical
https://www.ophthalmologytimes.com The early results of the phase 1/2a PIONEER gene therapy study showed that the combination of a new treatment, gene therapy and a medical device designed to treat end-stage non-syndromic retinitis pigmentosa (RP), was well tolerated....