A Scottish butterfly girl’s life has been confirmed as a miracle cure for a brutal situation.
A miracle new treatment approved yesterday will change the life of Isla Grist, a butterfly girl who suffers every day.
Scotland legend Graeme Souness’ wee pal Isla; At the age of 15, I was suffering from Epidermolysis Bullosa, which causes the skin to become red and watery at the slightest touch. In January, Souness, who is a close friend of Isla and her family, pleaded with health chiefs to help her by fast-tracking a new treatment that could ease her pain.
Their wishes came true when the Scottish Medicines Consortium approved the use of Filsuvez gel as the first licensed treatment in Europe. Filsuvez is a sterile gel for EB-related lesions in patients six months of age and older.
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It was approved for an initial three-year period through the SMC’s late-stage orphan drug pathway while additional safety and efficacy data were collected. Graeme befriends Isla and her family as he and his father Andy raise funds for the condition.
Royal Marines Last year I swam the English Channel with Graeme and Andy to raise funds for vital research into better treatments.
Graeme, A new challenge from Andy and the team is expected to be announced later this month.
Isla calls them her “heroes” and recently said: “Graeme is kind; Very kind. It has a bit of a hard shell, but it’s soft inside.”
Isla’s dad Andy, from Inverness, asked the SMC to make a quick decision six months ago: “Any topical cream or liquid that makes the skin stronger quickly has a big impact.
“I’ve seen the data from the trials and it’s great. We are very excited.”
As Isla’s skin was permanently covered in bandages, her skin was red and bleeding.
On average, she is hospitalized every two or three months. She has more than 60 general anesthetics, including one to stretch her throat, which does not affect the outer skin, so she can swallow food.
She has had 15 operations on her hands – one of the most painful of the many because her fingers are bent together.
The Pride of Scotland Award winner changes her clothes three times a week and uses ointments to treat the open, weeping wounds that cover her small body.
The procedure can take up to five hours. Isla said: “It hurts when I apply the cream.
“I don’t know if there is a way to describe the pain I always feel. It is at a high level. It’s a constant. itchy I shivered. Sometimes it feels like your body is on fire.”
Filsuvez gel treatment, made from the bark of a tree, in one trial showed complete wound closure within 45 days for many patients and reduced pain when changing dressings. In some patients, the body surface area covered by lesions is reduced by half.
Patients with dystrophic EB, like Isla, suffer from constant pain from open wounds and loss of vision; Increased risk of scarring and skin cancer. Most patients rarely survive beyond the age of 30.
Tony Byrne, Head of EB Charity Debra, said: “People living with all types of EB are in desperate need of treatments that can positively manage their symptoms and improve their overall quality of life. So the intersection and the first treatment for patients is very encouraging. Dystrophic EB was recommended by NICE in September last year and is now recommended by the SMC and the treatment is available across the UK.
“On behalf of the EB community, we thank Chiesi and the SMC for facilitating this approval and all our members who supported the application. “There is still much work to be done to ensure that approved treatments are available for all types of EB, but this is a promising first step.
“We hope it gives us hope today and will help with future therapies.”
Filsuvez and Peter Marinovich, director of Stanford Medicine’s Blistering Disease Clinic, said, “Wounds heal faster, but more importantly, they stay closed.”
Tony Byrne, CEO of DEBRA (Dystrophic Epidermolysis Bullosa Research Association) UK, said the announcement was very encouraging for the EB community, as David Garzón Lafuente, head of rare diseases company Chiesi UK&I, emphasized. A “deep commitment” is needed to support people living with a rare disease.
The gel should be available to eligible patients in September.
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