Wonder drug breakthrough for little Amalia

Photo: Darren Robinson

STAYING positive . . . mum Saima Javed and Amalia.

IT was a new year and a new lease of life for three-year-old Amalia Amer, who has been told wonder drug treatment to help her will be made available on the NHS.

Amalia’s family joined a national campaign urging the National Institute for Health and Care Excellence to permit access to the revolutionary Vimizim treatment. The Werneth youngster has Morquio Syndrome - a genetic disorder in which the body doesn’t make enough of an important enzyme.

Sufferers struggle to break down substances in their system and heart, lung, sight, hearing and dental complications can result - as well as skeletal abnormalities and breathing problems.

Amalia is one of only around 105 people with the condition in the UK, the average life expectancy of whom is only 25.

Her mum Saima Javed and dad Amer Zaman joined a year-long nationwide campaign for access to the new treatment, which can help to alleviate symptoms and improve quality of life. The family wrote to NIHCE, local MPs, NHS England and members of the Government about Amalia’s condition and how the enzyme-replacement therapy could improve her quality of life. Saima also took part in a sponsored skydive.

NIHCE has now issued final guidance recommending Vimizim for those with Morquio A in England, if delivered under strict conditions.

Saima said: “We are over the moon with the announcement. This is the day we have been waiting for since Amalia was diagnosed in June but for many it has been a rollercoaster ride for years. To be able to tell Amalia that she may be able to get the medicine she needs soon was fantastic.

“It will make such a difference to her.”

Amalia is now waiting for an assessment in January, before hopefully starting treatment in the next few weeks.

The drug has improved individual stamina and energy and allowed children to continue growing taller than untreated individuals. Children have required fewer operaions and are less reliant on wheelchairs and mobility aids.

The announcement came a month after Amalia returned home from a 10-day hospital stay following surgery to stabilise her spine after her condition worsened. She is spending four months with a metal brace around her head and spine while her bones fuse. It is hoped the procedure will allow her to continue walking and moving as normal.

Saima added: “We were terrified when she first went for the operation and it was a shock to see her in the halo for the first time.

“It really made us realise the magnitude of her condition and how her life would be affected without treatment.”