Home » Lifestyle » Toddler becomes first child in UK to have gene therapy cute fatal disorder – but it came too late to save older sister | The Sun

Toddler becomes first child in UK to have gene therapy cute fatal disorder – but it came too late to save older sister | The Sun

02/15/2023

A TODDLER has become the first child in Britain to have gene therapy to cure a fatal disorder — but it has come too late to save her older sister.

Teddi Shaw, who is 19 months old, had the treatment for metachromatic leukodystrophy (MLD), which destroys the nervous system and organs.

But it will not work for her three-year-old sister, Nala, as her condition is already too advanced.

Initially, the therapy, called Libmeldy, had a list price of £2.8million, but is now being offered at a discount to the NHS.

It halts MLD by inserting healthy copies of a faulty gene into the patient’s stem cells.

Parents Ally, 32, and Jake, 29, of Northumberland, said they were grateful Teddi now had a chance of a normal life.

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But they added: “Being told our daughter Nala wasn’t eligible for treatment was heartbreaking.”

"We can only hope that, one day, a treatment becomes available for all stages of MLD, and we feel strongly that it should be added to the newborn screening test to save more families from having to go through this heartache."

She added: "Teddi is doing absolutely brilliant.

"She is walking, running, a chatterbox – absolutely no signs so far of MLD.