A toddler becomes the first to be treated by the NHS with a new gene therapy costing £2.875 million.
But for 10-month-old Teddi, there was hope. The disease had not yet affected her and so she became the first patient treated on the NHS with a new life-saving gene therapy, called Libmeldy, which must be given before the disease has caused irreparable damage.
Teddi and her mum moved into hospital for the duration of the treatment while Jake, a carpenter, was home in Northumberland looking after Nala. What is remarkable is that this is a one-off treatment, with the hope that it provides a permanent fix for MLD. "We have had almost nothing to offer families with this condition for decades. Instead of many years of terrible neurodegenerative disease, we have the potential for a full life, lived healthily."Teddi's parents, along with other MLD families and the doctors who treat them, are campaigning to have it screened for at birth. In the UK, babies are given a heel-prick blood test which screens for nine genetic conditions, such as cystic fibrosis - but it does not currently include MLD.
One reason why the price tag is so high is to cover the costs of developing and producing the drug. The price paid by the NHS for this one-off treatment has to be set against the cost of treating children with MLD as they gradually become completely dependent, tube-fed and lose all their senses. And then, there is the suffering endured by patients and their families.
"You feel like you're grieving from the very start because your child is disappearing almost in front of your eyes," says Ally.
México Últimas Noticias, México Titulares
Similar News:También puedes leer noticias similares a ésta que hemos recopilado de otras fuentes de noticias.
Mental health charity in Tamworth facing closure over funding pressuresChanges Tamworth, which helps multiple NHS patients, says a lack of funds threatens its future.
Leer más »
The Crisis at the NHS, in Eight ChartsIn England, 7.1 million people were left waiting for nonemergency medical treatment. Eight charts show how the U.K.’s government-run National Health Service struggled to care for a nation.
Leer más »
Dentists in NI lose money on most NHS treatments, BDA saysPreliminary figures suggest carrying out some NHS treatments costs dentists money, the BDA warns.
Leer más »
The success of NHS England’s Innovative Medicines Fund will depend on its operational details - Nature MedicineThe success of NHS England’s Innovative Medicines Fund will depend on its operational details. Correspondence from Aris_Angelis and colleagues LSHTM.
Leer más »
Steven Spielberg Says Tom Cruise ‘Saved Hollywood’s Ass’Steven Spielberg says Tom Cruise may have saved the entire theatrical industry with ‘Top Gun.’
Leer más »