Experimental medicine shows the possibility of treatment of young patients with rare form of ALS
Sandy Verma May 23, 2025 04:25 PM

A team of American researchers said on Friday that treatment from practical drug has shown significant improvement in young patients with rare form of amiotrophic lateral sclerosis (ALS) – a progressive neurodynative disorder.

ALS, also known as Lu Gehrig Disease, is a rare disorder that affects nerve cells in the brain and spinal cord, causing loss of motor neurons to lead to movement, balance, coordination, and possibly difficulty in breathing.

While experimental remedies have slowed down the disease so far or have stopped its progression, new remedies have shown that functional damage in young patients can be reversed. Neurologist and scientist Neel Sayer at the University of the University said that the new treatment can be reversed. ” Not.

But, “What we have seen in a patient is truly unprecedented functional improvement. It is surprising and deeply motivated for us, for the ALS research community, but also for the community of ALS patients,” he said.

Data from 12 patients – All were treated with novel therapy for a rare form of ALS caused by genetic mutation in a gene called Fus – was presented in a case series published online by Schnider in The Lancet, although these gene mutations are responsible for only 1-2 percent cases of ALS, they become teenagers and young people who are responsible for only 1-2 percent of ALS. Start in adults. In patients with these mutations, toxic fus proteins accumulate in motor neurons that control the patient’s muscles, eventually killing neurons.

Two of the published case series showed remarkable response to the experimental therapy, Ulefnerson, which was developed by Schneider in collaboration with the California -based Ionis Pharmacuticals.

A young woman, who has received therapy injections from the end of 2020, has achieved the ability to breathe without any assistance and without the use of ventilator, which was first lost due to ALS. She has lived longer with this disease compared to any other known patient suffering from this teen-adolescents of fus als.

The second patient, who was a person in the mid -30s, was symptomatic when he began treatment, but electrical activity tests indicated that the symptoms were likely to emerge soon. In a three-year continuous treatment with experimental drug, the person has not yet developed any symptoms of Fus-Aals and has improved abnormal electrical activity in his muscles. Overall, after six months of treatment, the series patients experienced a deficiency of up to 83 percent in a protein called neuroflamant lights.

“These reactions indicate that if we interfere in time and reach the right goal at the right time during the disease, it is not only possible to slow down the progression of the disease, but it is actually possible to reverse some functional disadvantages,” Schnider said. Although the patients with most other symptoms of the series did not survive their aggressive disease, Schyider said that many people said “many people said to be a clear form from treatment. As a result, they survived for a long time. ”

The case series also showed that the drug is safe and well tolerated, no serious adverse incidents to the drug have occurred. After the earlier results, the global clinical test of the drug is now in progress. “Now we are eagerly waiting for the results, which we hope that we hope to be Elefanessen,” said Shnnordidars.

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