Spain has begun using a new therapy that may bring cautious optimism to a small group of people living with amyotrophic lateral sclerosis.
The disease, better known as ALS, progresses quickly and affects the nerve cells that control voluntary movement.
For years, treatment options have been limited and mostly focused on slowing symptoms.
Now doctors in Catalonia have started administering a drug that researchers believe could change how a specific genetic form of ALS develops.
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A new medical step
At the Germans Trias i Pujol University Hospital in Spain, specialists have started treatment with a drug called Tofersen.
The therapy has been given to a 44-year-old patient diagnosed with ALS linked to a mutation in the SOD1 gene.
This genetic variation accounts for roughly 2 percent of ALS cases and is among the most common inherited forms of the disease.
The European Medicines Agency approved compassionate use of the drug in 2024, and Spain’s Ministry of Health recently authorised and funded its use in the public healthcare system.
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What research shows
International clinical studies suggest the drug may help slow the functional decline experienced by patients with SOD1-related ALS.
Researchers observed that some patients maintained breathing capacity and muscle strength longer than expected.
In extension phases of the trials, about a quarter of participants showed early improvement in clinical measurements of daily function.
Tofersen works by targeting the genetic mutation believed to drive the disease process in this subgroup of patients.
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How the treatment works
The medication is delivered through injections into the spinal fluid. The first stage involves three doses given every two weeks, followed by monthly treatments.
The therapy requires coordination between neurologists, anesthesiologists and hospital pharmacy teams.
ALS remains one of the most aggressive neurodegenerative diseases. It gradually destroys motor neurons in the brain and spinal cord, leading to muscle weakness, paralysis and eventually respiratory failure.
Sources: Infobae
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