Medications
Here are some examples of medications for managing ataxia symptoms. All of them must be prescribed by a neurologist. The list below is for reference only.
MEDICATIONS FOR ATAXIA APPROVED BY ANVISA FOR MARKETING IN BRAZIL
01
SKYCLARYS (Omaveloxolone)
Laboratory: Biogen
Indication: Friedreich's Ataxia (patients 16 years or older)
Approval by ANVISA: 04/11/2025
Price of the medicine defined in CMED: Pending
CONITEC Opinion for incorporation into the SUS: Pending
Ministry of Health decision on incorporation into the SUS: Pending
Comments: SKYCLARYS (Omaveloxolone) was approved in February 2023 by the FDA specifically for Friedreich's ataxia. It was the first FDA-approved ataxia medication in the world (specifically for Friedreich's ataxia). This important drug was produced by Reata Pharmaceuticals, which launched the drug under the trade name SKYCLARYS on February 28, 2023. That same year, Reata was acquired by Biogen. Briefly, OMAV (or SKYCLARYS) works by improving mitochondrial energy-producing function within cells. Dr. Susan Perlman (an ataxia specialist) highlighted in a webinar the excellent work Reata Pharmaceuticals did with the FDA to demonstrate the benefits and safety of OMAV, thus speeding up the time to approval of this drug as much as possible. She mentioned that "this is a model that other pharmaceutical companies should strive to follow." For example, Reata used natural history studies of Friedreich's ataxia to supplement data from the drug's clinical trials. It's worth noting that a clinical trial of OMAV was also conducted in Brazil, supported by ABAHE and with the participation of brilliant Brazilian researchers, as part of the global study.
MEDICATIONS FOR MANAGING COMMON SYMPTOMS IN ATAXIAS
The medications listed below do not cure or slow the progression of ataxias, but they help manage specific symptoms, improving patients' quality of life.
1 – Neuropathic Pain / Dysesthesias
Gabapentin, Pregabalin, Duloxetine, Amitriptyline, Amantadine (Mantidan)
2 – Spasticity
Muscle relaxants such as Lioresal (Baclofen) and Zanaflex (Tizanidine) may be prescribed. CBD oil (Cannabidiol) may also help.
See Note 3 in Additional Information for Baclofen.
See Note 7 in Additional Information for Cannabidiol.
3 – Cramps and Muscle Spasms
Lioresal (Baclofen) and Zanaflex (Tizanidine).
See Note 3 in Additional Information for Baclofen.
4 – Nystagmus
Acetazolamide (Diamox), 4-aminopyridine (4-AP), Baclofen, Carbamazepine, Clonazepam (Klonopin), Gabapentin (Neurontin), Isoniazid, Memantine.
See Note 2 in Additional Information for 4-AP.
5 – Dizziness and Vertigo
Acetazolamide (Diamox), 4-aminopyridine (4-AP), Baclofen, Clonazepam, Flunarizine, Gabapentin (Neurontin), Meclizine, Memantine, Ondansetron (Zofran), Scopolamine.
See Note 3 for Baclofen and Note 4 for Meclizine.
6 – Gait Imbalance
Amantadine, Buspirone (Buspar), Riluzole (Rilutek), Dalfampridine, 4-Aminopyridine (4-AP). In addition to medications, exercise (gym), water aerobics, yoga, Pilates, and especially neurofunctional physical therapy are recommended to improve coordination, balance, strength, and reduce fall risk.
See Note 1 for Riluzole and Note 2 for Dalfampridine and 4-AP.
7 – Fatigue
Amantadine, Coenzyme Q10, Idebenone, Eudok (antidepressant). It’s also important to rest as needed and maintain good nutrition to support energy metabolism.
See Note 8 for Amantadine.
8 – Excessive Daytime Sleepiness
Modafinil (Provigil), Armodafinil (Nuvigil)
9 – Memory Issues
Cholinesterase Inhibitors, Memantine (Namenda)
10 – Muscle Weakness
Creatine (up to 3g/day)
11 – Dysarthria (Slurred Speech)
Speech therapy (with a speech-language pathologist) is recommended.
12 – Dysphagia (Swallowing Difficulties)
Pyridostigmine and speech therapy are strongly recommended. Liquid thickeners may also help.
13 – Dystonia
Botulinum Toxin
14 – Cardiomyopathy (Friedreich’s Ataxia)
Coenzyme Q10, Idebenone
15 – Urinary Incontinence
Oxybutynin, Botox injection, Tamsulosin Hydrochloride 0.4mg
16 – Constipation
Metamucil, Miralax, Magnesium Citrate. Also recommended: stay well hydrated, eat prunes daily, and maintain a high-fiber diet.
17 – Tremors (Parkinsonism)
Prolopa, Levodopa
See Notes 5, 6, and 8 in Additional Information.
18 – Restless Legs Syndrome
Pramipexole, Gabapentin
19 – Anxiety
Cannabidiol
See Note 7 in Additional Information.
20 – Diplopia (Double Vision)
Prism glasses, Botox injection, and in more severe cases, surgery.
21 – Depression
Citalopram, Fluoxetine, Paroxetine, Sertraline, Eudok, Cymbalta (Duloxetine), Elavil (Amitriptyline)
Last updated: July 28, 2023
Sources: Testimonies from patients with ataxia in the NAF Support Group on Facebook and the ABAHE Ataxia Support Group on WhatsApp, and the list of medications for ataxias published on the NAF website (see link above).
Talk to your neurologist about medications
Some medications for ataxia symptoms work well for some people and not for others. Dr. Henry Paulson explained in a NAF webinar in March 2023 that "the only way to know if a medication works for someone specifically is to try it, with proper supervision." If a medication doesn't work—for any symptom—an alternative should be sought.
Dr. Paulson made the important point that "patients with ataxia need to talk to their doctor and try medications (in a controlled manner) until they find one that works well for them, without significant adverse effects." Dr. Paulson recommended bringing a "reference medication list" published on the NAF website (link below) to DISCUSS WITH THE NEUROLOGIST. Ask them directly, "Doctor, do you think any of these medications could help me?"
Keep in mind that ataxia is a rare disease, and that a physician will typically see few (perhaps none) patients with ataxia in their practice. Therefore, Dr. Paulson considers it important to "interact with the physician," who can and should also learn from the ataxic patients they see. This interaction and exchange of information with the physician helps the patient, helps the physician, and also helps others they will see in the future.
Additional information on ataxia medications
1, Riluzole
Dr. Susan Perlman reported in a NAF webinar that Riluzole (a medication approved by the FDA for Amyotrophic Lateral Sclerosis (ALS)) has shown positive results in some patients as an off-label medication to treat ataxia symptoms. Based on studies conducted in Europe, it has purported neuroprotective effects (protecting the nerves in the cerebellum). It is a glutamate modulator. However, a known side effect of Riluzole is dizziness . Therefore, if a person already experiences dizziness due to ataxia, Riluzole may worsen this symptom, which in turn affects balance. Dr. Perlman said it is not common, but she has seen patients with this side effect in her clinical practice. Therefore, it is necessary to evaluate in these cases whether or not it is beneficial to use the medication.
Note: Troriluzole
In addition to riluzole, which is already on the market, there is Biohaven Pharmaceuticals' Troriluzole (clinical trial BHV-4157), a sort of upgrade to riluzole, which is seeking FDA approval. Troriluzole is metabolized into riluzole in the body, making riluzole the active ingredient. The difference is that Biohaven's new formulation aims to be more effective.
LEARN MORE ABOUT TRORILUZOLE (Biohaven Pharmaceuticals)
2. AMPYRA® (Dalfampridine) and 4-AP (4-Aminopyridine) for Ataxia
Dr. Susan Perlman stated in a webinar that Dalfampridine is a drug that helps improve gait in patients with Multiple Sclerosis (MS), a condition that shares many symptoms with ataxias. She explained it could be a good option to try under medical supervision, of course (speak with your doctor, assess whether it’s worth trying—each case is different). There is also a related medication, 4-AP (4-aminopyridine), whose active ingredient is Dalfampridine. 4-AP appears to offer benefits to patients with episodic ataxia type 2 (EA2) and to those with spinocerebellar ataxia type 27B (SCA27B).
There is some concern about potential side effects, especially in the case of accidental overdose, such as the risk of seizures. However, if the person stays within the safe dosage limits (no more than 20 milligrams per day), the risk of seizures and other side effects is minimal. Naturally, those already at risk of seizures for other reasons should exercise great caution. According to reports in support groups, Dalfampridine may improve gait balance in certain individuals. It belongs to a class of medications known as potassium channel blockers, which work by strengthening signals sent from the brain through nerves that have been damaged in multiple sclerosis. Although it is an MS drug, many people with ataxias also use it, and there are even Facebook support groups specifically discussing its use.
IMPORTANT – Dr. Perlman mentioned that the only two drugs currently recognized by the American Academy of Neurology for off-label use in ataxia are Riluzole (FDA-approved for ALS – Amyotrophic Lateral Sclerosis) and Dalfampridine (AMPYRA), approved in 2010 for MS.
3. Baclofen
Baclofen is a medication prescribed by neurologists to reduce spasticity and help relax muscles to relieve pain, muscle spasms, and cramps, which can affect some patients with ataxia. According to Dr. Susan Perlman (ataxia specialist), it is best to take this medication at night before sleep. During the day, rather than medication, try stretching and physical exercises to relax muscles. Note: although sometimes confused, spasticity is different from muscle rigidity, and the medications used to treat them are quite different.
4. Medications for Dizziness and Vertigo
Dr. Perlman explained in a NAF webinar that dizziness is a challenging symptom to manage and may have multiple causes (some unrelated to ataxia, such as blood pressure fluctuations). If the dizziness comes from the brain’s connection to the inner ear, medications like Meclizine or Bonine may help. In some ataxia-related dizziness, Dalfampridine (off-label use) has been prescribed and shown benefits in some cases. If dizziness is caused by abnormal eye movements (nystagmus), other medications may help reduce these movements, which can contribute to dizziness. In short, dizziness has many causes, and a physician should be consulted to determine the best approach.
5. Levodopa and Prolopa
Levodopa increases the amount of dopamine in the body, a neurotransmitter responsible for communication between neurons and control of body movement and coordination. It revolutionized Parkinson’s treatment. Dr. Susan Perlman (ataxia specialist) explained in a webinar that "there are different types of tremors with different causes," and that for a specific group of tremors associated with ataxias, there’s evidence that Levodopa may help. Naturally, this medication must be prescribed and dosed by a neurologist. Other Levodopa-based drugs may also help with ataxia-related tremors, such as Prolopa (levodopa + benserazide hydrochloride). Always use with medical supervision!
6. Azilect
In a NAF webinar, Dr. Perlman stated that Azilect is prescribed to treat Parkinson's disease in adults and may help with tremors in ataxia. Azilect acts on areas involving the basal ganglia, the brain region most affected in Parkinson’s. Since the cerebellum communicates with the basal ganglia, and some ataxia-related tremors result from issues in this communication, drugs like Azilect, Sinemet, and Carbidopa-Levodopa may help with tremors not only in Parkinson’s but also in ataxia.
7. Cannabidiol (CBD)
Although it does not cure or slow ataxia progression, medical cannabis can help relieve certain symptoms. Cannabidiol oil (CBD)—along with other compounds like cannabigerol or THC prescribed in precise ratios by a neurologist—can help with sleep, spasticity, neuropathic pain, and anxiety. Medical prescription is required.
8. Amantadine
Dr. Susan Perlman explained in a webinar that Amantadine (sold in Brazil as Mantidan) is one of the oldest off-label drugs used in ataxia to help with tremors and fatigue. Amantadine was originally developed as an antiviral, but was later found to improve mobility in Parkinson’s disease. It is FDA-approved to treat primary Parkinson’s disease and symptoms of secondary Parkinsonism. For years, it has also been used off-label to treat ataxia-related tremors and fatigue. The drug is considered safe, including for children, but it has side effects, so patients should talk to their neurologist and test it over time to balance benefits and risks.
9. Chlorzoxazone (for SCA1)
Chlorzoxazone is a muscle relaxant. Dr. Perlman mentioned in a webinar that researchers have shown interest in its potential neuroprotective effects, but that it should be used under close medical supervision.
Note from MG: Some studies report that the combination of Chlorzoxazone with Baclofen may be beneficial for spinocerebellar ataxia type 1 (SCA1), when used in very specific dosages. However, studies are still preliminary, and more testing is needed to determine effective dosages that improve symptoms without causing toxicity or affecting muscle tone excessively (which may worsen balance).
See: https://pubmed.ncbi.nlm.nih.gov/33151010/
See: https://www.ataxia.org/scasourceposts/a-new-use-for-old-drugs/
NAF (National Ataxia Foundation) Webinars on Ataxia Medications
The videos are in English, but you can activate subtitles and enable translation into Portuguese.
Link to NAF YouTube channel
Download the .pdf version - NAF AAC 2025 - Medications and Diet for Ataxia.pdf
NAF (National Ataxia Foundation) English paper presented at AAC (Annual Ataxia Conference) 2025.
Presentation: Dr. Susan Perlman
Medications & Diet for Ataxia
Day 1 - AAC 2024 - NAF ( National Ataxia Foundation ) - Liana Rosenthal, MD, PhD (video posted Feb 6, 2025)
FDA Drug Approval
The FDA (US Food & Drug Administration) is responsible for protecting public health in the United States by ensuring the safety and efficacy of drugs, therapies, and biological products for human and veterinary use. In Brazil, ANVISA (National Health Surveillance Agency) plays a similar regulatory role. The FDA (and also ANVISA) approval process for new drugs is highly technical, rigorous, and time-consuming and can be found on this page . Recently, the FDA has shown a bit more flexibility in reviewing the approval of drugs and therapies for rare diseases. Drugs for ALS (amyotrophic lateral sclerosis) and Alzheimer's disease have recently been approved based on data and studies that would not have been accepted 10 years ago (the rigor was greater, and the process slower).
Note: An " off-label " medication is one that is used for one condition but has been approved for another. For example, Riluzole is FDA-approved for ALS (Amyotrophic Lateral Sclerosis), but is used "off-label" by some patients with ataxia (a condition for which the medication has not yet been formally approved), with a proper medical prescription.
Drugs requiring caution in patients with mitochondrial diseases (PolG gene)
It's understandable that patients with rare diseases with no cure may try various medications (many off-label , not yet FDA-approved for the disease in question), as well as various therapies. Regarding this, the first recommendation is to always consult with your doctor. Medications have side effects, and some can interfere with others. In the specific case of the various syndromes and mitochondrial diseases caused by mutations in the PolG gene, patients are advised to exercise CAUTION when trying medications, as some can have side effects that can worsen symptoms!
For example, valproic acid (Depakene) , prescribed for epilepsy, is contraindicated (see package insert) for patients with Alpers-Huttenlocher syndrome and others with significant liver dysfunction. The same is true for divalproex sodium (Divalproex, Depakote) , usually prescribed for patients with manic episodes associated with bipolar disorder and for migraine prevention. These medications should be avoided by patients with mitochondrial dysfunction associated with the PolG gene due to the risk of accelerating or worsening liver dysfunction.
See
https://www.ncbi.nlm.nih.gov/books/NBK26471/
ALL MEDICATION MUST BE PRESCRIBED BY DOCTORS.
Medications that can cause ataxia!
In addition to hereditary ataxias, ataxia symptoms can be acquired for a variety of reasons, such as alcohol abuse, heavy metal poisoning, vitamin E and B12 deficiency, gluten intolerance, strokes, neuroimmunological problems caused by diseases such as MS (Multiple Sclerosis), and, under certain conditions, also by certain medications. A typical example is "Benzos": "Prolonged use of some barbiturates such as phenobarbital (an anticonvulsant) and sedatives such as benzodiazepines ("Benzos") can cause ataxia, as can anti-epilepsy drugs (such as phenytoin) and some types of chemotherapy. Source: Mayo Clinic." For this reason, medication should never be taken without a prescription, and anyone with ataxia should ALWAYS disclose this condition at any medical appointment, as the doctor may prescribe a drug that may not be recommended for someone who already has ataxia.
DISCLAIMER: The information provided is for educational purposes only and is not intended as medical advice.
Always seek the guidance of a qualified physician or other licensed health professional before starting, changing, or stopping any medication or treatment. All medications must be prescribed by a doctor and may have potential side effects..