Latest Breaking News On - டேவிட் டேவிட்சன் - Page 1 : comparemela.com

Dive Brief: A committee that advises Europe s drug regulator is recommending that a gene therapy developed by Bluebird bio for a rare, often deadly disease be approved for market. The disease, known as adrenoleukodystrophy or ALD for short, is caused by genetic mutations that lead to a toxic buildup of molecules in the brain and spinal cord. In clinical testing, Bluebird s therapy, now called Skysona, has shown positive effects on cerebral ALD  the most common and severe form of the disease  with the vast majority of treated patients staying alive and free of major functional disabilities. The European Medicines Agency is now advising the European Commission to authorize Skysona for the treatment of early cerebral ALD in children with certain genetic mutations who don t have a sibling that can be a match for hematopoietic stem cell donation. Bluebird says a final decision is expected in mid-2021.

United kingdomUnited statesDavid davidsonEuropean unionEuropean medicines agencyEuropean commissionDrug administrationEuropean leukodystrophies associationDive briefThough bluebirdGuy albaGene therapyஒன்றுபட்டது கிஂக்டம்ஒன்றுபட்டது மாநிலங்களில்டேவிட் டேவிட்சன்ஐரோப்பிய தொழிற்சங்கம்