Ataxia still has no cure, but it can be treated!
There is currently no cure for hereditary ataxias or for most acquired ataxias.
However, science is advancing and there is a lot of research underway in search of medicines and therapies that can cure or at least reduce the progression of the disease.
Proof that science is advancing was the approval by ANVISA on April 11, 2025, for the marketing in Brazil of the first medication for ataxias, SKYCLARYS. (from the pharmaceutical company Biogen) for Friedreich's ataxia (FA). This was an important milestone for ALL ataxia patients, not just the "Fredericos," as FA patients are affectionately called.
Although there are no medications to cure ataxias, they can (and should) be treated. The same is true for AIDS, which also has no cure yet but can be treated, as well as for several types of cancer and countless other diseases.
Diagnosis matters
Some ataxias have a mild or controllable course when diagnosed early. With a rapid diagnosis and timely treatment, it is often possible to prevent permanent damage to the nervous system and, in many cases, stop the onset of symptoms.
One example is AVED (ataxia with vitamin E deficiency), an inherited disease with autosomal recessive transmission caused by mutations in the TTPA gene, which encodes a protein responsible for the transport of vitamin E. When left untreated, AVED can lead to progressive degeneration of the cerebellum and posterior spinal tracts, resulting in symptoms such as imbalance, dysarthria, and peripheral neuropathy. However, if the diagnosis is made early — for example, in asymptomatic relatives or at the first clinical signs — and the patient begins vitamin E supplementation in high doses (under medical supervision), it is often possible to prevent or stabilize the disease’s progression and even reverse early symptoms. For this reason, AVED is considered one of the few potentially treatable hereditary ataxias, highlighting the importance of early genetic and biochemical screening in suspected cases of ataxia, especially in children or adolescents.
There are also acquired (non-genetic) ataxias in which early diagnosis and proper treatment can lead to an almost complete remission of symptoms or, at the very least, prevent disease progression. Gluten ataxia is one of the best-documented examples, but there are other treatable causes as well.
Treatment strategies
A neurologist may prescribe medications to treat symptoms. However, in addition to medication, there are other therapies and resources that can help manage symptoms. If new symptoms appear, the patient should seek medical advice and be referred to the appropriate specialist.
These measures allow ataxia patients to have a better quality of lifeand make them more independent in managing a wide variety of daily activities for as long as possible. For example, the ophthalmologist may prescribe glasses with prism lenses that can help with double vision (diplopia), a common symptom in some ataxias.
Multidisciplinary team
It is important that anyone with ataxia is monitored by a multidisciplinary team, coordinated by a neurologist , including, for example (depending on the patient's needs during the course of the disease), an ophthalmologist, neurofunctional physiotherapist, occupational physiotherapist, speech therapy professionals, nutritionist, psychologist, etc.
PHYSIOTHERAPY IS VERY IMPORTANT FOR THOSE WITH ATAXIA.
Neurological physical therapy is crucial, as are speech therapy, occupational therapy, and physiatry, as well as psychological support with individualized and group sessions. Yoga, Pilates, and core strength exercises are also recommended.
Many types of ataxia can affect the coordination of the muscles involved in speech, swallowing, and breathing in some patients. Speech-language pathologists can help patients with ataxia with symptoms of dysarthria (slow, slurred, or inarticulate speech) and dysphagia (difficulty swallowing, which can increase the risk of choking and aspiration pneumonia).
Learn more about the importance of speech therapy for patients with ataxia on this page .
Occupational Physiotherapy (OT) is also important to help patients preserve their independence, improve their quality of life and adapt their daily lives to the limitations of the disease, which can be progressive.
DON'T GIVE UP.
With ataxias there are good days and bad days, but you are not alone!
Advances in gene therapies and other ongoing approaches represent promising possibilities for all ataxia patients and will mean, in the increasingly near future, the possibility of definitive treatment for this group of diseases.
Photo: Renata, Abahe volunteer, diagnosed with SCA3 ataxia, with her "Fight as a Woman!" shirt.
Image posted with the patient's permission.