Sunday Express praised in Motor Neurone Disease breakthrough | UK | News
A new groundbreaking treatment for motor neuron disease (MND) is approved for use in the UK and offers new hope after decimal despair.
The drug called QALSody (Tofersen) is the first person to directly target the genetic cause of the disease in the UK. In almost 30 years, the first new treatment for MND is greeting as a “real breakthrough”.
The new drug will help with a rare MND type caused by a mistake in the SOD1 gene, which is thought to affect less than 60 people in the UK. However, experts say that the breakthrough shows the potential of a new gene -targeted treatment, which is currently called in other Mnd forms that offer hopes to thousands of people.
Professor Chris McDermott, a leading consultant neurologist and Sheffield Mnd Maintenance Center co -director, said: “This drug is a Gamechanger for many patients who noticed one step change in the progress of the disease.
“This technology is an important milestone for all MND patients and is now used to target other key genes and proteins for other MND forms in trials.
“This means that we believe that we believe that we should do a meaningful treatment for everyone who has this situation over the years instead of decades, instead of a very exciting time and the future for MND. In addition, we do not give up a treatment.”
MND attacks the nerves that control the movement, and slowly paralyzes patients until they can no longer walk, speak, swallow or even breathe.
It is diagnosed with approximately 2,000 people every year in the UK. Approximately 5,000 people live with illness at any time and claim the lives of six people every day.
Life expectations are short: Most of them die within two to five years after the start of symptoms.
Campaigns, despite the major public pressure of the UK is behind in offering new therapies, he says.
The news follows the Fund to improve a Sunday Express’ Fund ‘MND’ campaign in partnership with the MND Association to demand serious government actions.
The campaign succeeded after months of powerful stories and relentless lobbying. Later – Prime Minister Boris Johnson promised £ 50 million for the targeted MND research – now, now it is used to create a special MND Research Institute that accelerates treatments and treatment prey.
This last drug approval is proof that war is not in vain.
The three main UK MND Charity Authority – MND Association, MND Scotland and My Name’5 Doddie Foundation – welcomed the announcement and called the MND an important milestone for the MND community ”.
It was also welcomed by leading experts.
The money collected by Sunday Express was used for treatments that allow us to accelerate the drug development process in this hunting. “
Developed by the biotechnology company Biogen, Qalsody works by preventing the production of a harmful protein that causes nerve damage. In trials, compared to placebo, nerve damage reduced key marker levels by 55%.
Biogen’s General Manager of England and Ireland. Kylie Bromley said: “This confirmation is a real moment of hope for the MND community. We need to work with NHS to ensure that patients can access as quickly as possible.”
Despite the approval of the UK Pharmaceutical Regulator, Pharmaceutical and Health Products Regulatory Agency (MHRA), the drug is not yet available through NHS.
Before this happens, the National Health and Care Excellence Institute (NICE), which decides whether the treatments provide value for money, should be evaluated.
Since the SOD1 type Mnd is very rare, the trial data is limited. This makes it difficult for the beauty to approve, despite the helpless need. Campaigns Nice and NHS call England to show flexibility.
Last week, the MND community organized a strong protest in London and revealed a patient’s life -sized ice statue in a wheelchair outside the parliament.
The melting figure symbolized how fast time is exhausted for those living with the disease with the pleading of campaignists: iz We cannot wait. ”
MND received some of the most popular figures of England. Doddie Weir, the legend of Rugby, who borrowed his name and passion for fighting, died in 2022. Former footballer Stephen Darby spoke from his own struggle.
Families usually realize what is happening, while the disease is forced to follow the slowly disappearance of a loved one.
Motor Neuron Disease (MND): A fatal condition that attacks the nerves controlling muscles.
Life expectation: usually 2-5 years after symptoms start.
Effect of England: 2,000 new cases each year, 5,000 people living with illness.
Daily fee: Six deaths per day.
Inherited cases: Approximately 15% is genetic. SOD1 mutation affects approximately 2% worldwide, less than 60 in the UK.
Qalsody is already available in the USA and Europe. Mhra’s decision puts England on the map, but the campaignists emphasize that it is only the first step.
