Mackenzie Casella was a much-loved baby girl who didn't make it to her first birthday after being diagnosed with spinal muscular atrophy. 9News
"When we asked what that meant we were told it was a terminal diagnosis and she would likely pass away before she was eight months old," Mackenzie's mum Rachael Casella said.Mackenzie Casella was a much loved baby girl who didn't make it to her first birthday after being diagnosed with spinal muscular atrophy ."The impact that this new therapy is having on patients is incredible," Casella said.
More than 40 newborns with the devastating condition have been successfully treated with the game-changing therapy in NSW since 2019. SMA is an inherited neuromuscular disorder and a leading genetic cause of infant death in Australia, according to the Children's Medical Research Institute "We find that treated patients make a huge antibody response to the AAV delivery system and this has given us insights into the complexity of the human response to the AAV gene therapy," Logan said.
United Kingdom Latest News, United Kingdom Headlines
Similar News:You can also read news stories similar to this one that we have collected from other news sources.
Baby's tragic death leads to 'transformative' treatment breakthroughMackenzie Casella was a much-loved baby girl who didn't make it to her first birthday after being diagnosed with spinal muscular atrophy. 9News
Read more »
Beloved drinks line to vanish from shops, servo shelves nationallyIf you&x27;ve been thinking something&x27;s been missing from the servo shelves lately, you&x27;re not wrong.
Read more »
Beloved drinks line to vanish from shops, servo shelves nationallyIf you&x27;ve been thinking something&x27;s been missing from the servo shelves lately, you&x27;re not wrong.
Read more »
Baby gorilla survives traumatic birth at NSW wildlife parkA baby gorilla is now happy and healthy after he was fighting for life from a traumatic birth on the NSW South Coast. 9News
Read more »
Baby gorilla survives traumatic birth at NSW wildlife parkA baby gorilla is now happy and healthy after he was fighting for life from a traumatic birth on the NSW South Coast.
Read more »
Ash Barty welcomes first child with husband Garry KissickThe couple announced Barty&x27;s pregnancy in January, each sharing a photo of their dog, Origi, lying next to a pair of baby shoes via their respective Instagram accounts.
Read more »