The NSW state government will permanently fund screening for a rare but devastating genetic disorder with a high infant death rate, after a study conducted in Sydney showed early therapy could turn diagnosis from a “heart-sink” to a treatable condition.
Of the 29 babies with spinal muscular atrophy (SMA) who took part in a global trial of a new gene therapy, including four treated at Sydney Children’s Hospital, all reached 18 months and the majority achieved their developmental milestones, such as sitting independently and walking.
Hannah Devlin, Guardian UK Science Correspondent, reports that two US patients have recovered from intractable infections after being treated with a pioneering phage therapy involving genetically engineered bacteria-killing viruses.A 26-year-old man with cystic fibrosis, was approaching death after suffering a chronic lung infection. After being given the phage therapy, his infection cleared allowing him to receive a lung transplant and resume an active life.And, a 56-year-old man with severe arthritis, showed a remarkable recovery from a skin infection that was taking hold of his body and which had proved untreatable with conventional drugs. For more information see: https://www.inkl.com/news/phage-