The family of a little boy with a rare genetic condition who campaigned for the NHS to fund a miracle drug to prolong his life are hoping another life-saving drug will be approved.
Haris Khan, from Wythenshawe, was diagnosed with the devastating genetic condition Spinal Muscular Atrophy (SMA) at six-months-old.
The condition affects the nerve cells needed to control the muscles we use for moving, swallowing and breathing.
With no treatment available for SMA, specialist doctors told Haris' devastated family that he may only live for another three months.
One in 40 people are a carrier of the faulty gene and in the most severe cases, such as Haris, children rarely live beyond two years, making SMA the leading cause of death in babies and toddlers in the country.
But with the thought of losing their son impossible to comprehend, Haris' dad Shakeel and mum Renata started a campaign for NHS England to provide funding for Spinraza - a drug which has had phenomenal success in treating infants with SMA.
Read more of today's top stories
The campaign was a success and Haris had his first dose of the treatment in June 2019.
The now two-year-old receives the treatment in hospital once every four months.
Now his parents hope another potentially life-saving drug will be funded by NHS England which could prolong Haris' life for even longer.
Dad Shakeel told the Manchester Evening News it could even allow him to sit up or walk.
Onasemnogene abeparvovec - known commonly as Zolgensma - is a one-off treatment for SMA. The gene therapy medicine is given to babies as a one-off infusion.
The drug has been, and is still being, tested in several different clinical trials but so far has shown to be safe and well-tolerated by those treated.
It was approved for use in the USA and Europe for children under two after patients treated with the medicine were able to sit up, talk and walk.
Novartis Gene Therapies is conducting a clinical trial to see if the drug is effective for older children like Haris.
The trial is currently on hold but has provided 'strong interim results'.
The drug is being considered for approval by the National Institute for Health and Care Excellence (NICE) for NHS England use.
Experts held a committee meeting on February 10, with the outcome yet to be published.
Shakeel said the family are now just 'waiting and hoping'.
"My son is stable and we're enjoying family time but what we want to do now is be prepared", he said.
"If Haris can get this treatment, he can live longer and possibly stand and maybe even walk.
"It would also mean that we as a family could also have more children knowing that they can be successfully treated if they have SMA, as there is a one in four chance they will."
Shakeel said if the drug is not approved by NICE, he will consider starting another campaign.
In 2019 when the Khan family were campaigning for NHS England to fund Spinraza, the Wythenshawe community stepped up and showed tremendous support for them.
Every neighbour on their street had a poster in the window about SMA.
A number of celebrities also supported the campaign.
You can follow Haris' journey on his Instagram page here.
