NHS provides life-saving gene therapy treatment for the fatal disorder metachromatic leukodystrophy to baby for the first time.
In what is being described as a "breakthrough moment", the NHS has provided for the first time a life-saving gene therapy treatment for the fatal disorder metachromatic leukodystrophy to a baby.
How quickly the disease progresses and how long children with it live varies based on age when symptoms appear. Children with the late infantile type of MLD, which starts before 30 months and is the most common and the most rapidly progressing type, deteriorate rapidly and usually die between the ages of 5-8 years. Children with early juvenile MLD, which usually starts between 30 months and 6 years, have a life expectancy of 10-20 years after onset.
NICE emphasised that the recommendation prevailed only if the company provided the treatment "according to the commercial arrangement". The regulator also stated that atidarsagene autotemcel should be delivered in a 'highly specialised' service by a specialist multidisciplinary team. Professor Simon Jones, consultant in paediatric inherited metabolic disease, Manchester Centre for Genomic Medicine at Saint Mary's Hospital, and clinical director of NIHR Manchester clinical research facility at Royal Manchester Children’s Hospital, explained that MLD is a "progressive, life-limiting" condition and that prior to this metabolic disorder service being made available via the NHS, there were "no approved treatment options available".
The first child in the UK to receive the treatment outside of a clinical trial is now 19 months old, having started treatment when she was 12 months old. She is said to be a "happy and healthy toddler showing no signs of the devastating disease she was born with".
México Últimas Noticias, México Titulares
Similar News:También puedes leer noticias similares a ésta que hemos recopilado de otras fuentes de noticias.
First infant anonymously dropped off at Kentucky 'baby box' surrender locationThe first infant in Kentucky was anonymously dropped off at one of the state's “baby box” safe surrender locations. A 2021 law allows parents to surrender their newborns.
Leer más »
Star Wars: The Mandalorian Director Teases More Central Role for Grogu in Season 3A director of StarWars: TheMandalorian teases Grogu taking on a more central role in Season 3 of the series.
Leer más »
Gracie Hunt, First Daughter of the Kansas City Chiefs, Isn't Just a Nepo BabyLast night, the Kansas City Chiefs won the Super Bowl. The team owner's daughter Gracie Hunt spoke to us last week about beauty pageants and nepo baby discourse.
Leer más »
Stacey Dooley shares first video of baby Minnie'The loves of my life.'
Leer más »
NICU Nurses Capture Baby’s First Valentine’s DayFrom love bugs to kissing booth operators, these tiny tots might be this year’s most adorable Valentines. See the adorable images from their Valentine's Day photoshoot here.
Leer más »