There are also clinical trials for new treatments. Liver transduction of AAV-G6PC increases the long-term efficacy of treatment for glycogen storage disease type Ia [23]. Gene therapies with different strategies, including liver depot gene therapy, have been tested for the treatment of glycogen s...
There are also clinical trials for new treatments. Liver transduction of AAV-G6PC increases the long-term efficacy of treatment for glycogen storage disease type Ia [23]. Gene therapies with different strategies, including liver depot gene therapy, have been tested for the treatment of glycogen s...
The Strimvelis registry sets a precedent for the safety monitoring of future gene therapies. A unique, patient-focused design has been implemented to address the challenges of long-term follow-up of patients treated with gene therapy for a rare disease. Strategies to ensure data completeness and...
The global rare disease gene therapy market is categorized on the basis of type, indication, administration, distribution channel, and region. In terms of type, the market is bifurcated into non-viral vector gene therapy, and viral vector gene therapy. In terms of indication, the ma...
gene therapy development, and more. In light of the challenges faced with orphan drug development, bioinformatics and artificial intelligence (AI) tools have become essential for researchers, biotechs, and pharmaceutical companies developing rare disease therapies to accelerate drug discovery and vastly ...
The Latest research study released by CMI "Global Gene Therapy for Rare Disease Market" with 360+ pages of analysis on business Strategy taken up by key and emerging industry players and delivers know how of the current market development, landscape, technologies, opportunities, market viewpoint ...
Genethon develops innovative therapies for rare diseases of the eye, the liver, the immune system and for neuromuscular diseases.
A rare genetic disease that renders children without a functioning immune system from birth has been effectively cured by an experimental gene therapy. A study has reported on the first 10 children treated with the therapy, all of whom are now healthy.
Gene therapy project cures dogs of rare disease.(St. Louis Post-Dispatch)Hesman, Tina
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three to six times each week.