Treatments
Learn approved ALS/MND treatment options
Review status: Waiting for team review. An AI editorial check was completed on 2026-08-14 by claude-opus-5. It checks clarity, attribution and scope against the sources, and it is not a Compass team or clinical sign-off. Waiting for clinical review. Last updated 2026-08-14.
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Planning 3
- Review current treatment optionsAsk what approved treatments are available and whether any are relevant now.Why it matters: What is approved and available differs by country and changes over time, so it is worth asking directly rather than assuming.
- Prepare questions for your clinicianWrite down what you want to ask about benefit, side effects, and monitoring.Why it matters: These conversations move quickly, and written questions are what stops the important one being forgotten.
- Clarify access and next stepsCheck prescriptions, approvals, supply, and when follow-up happens.Why it matters: Delays are often administrative rather than medical, and they are easier to prevent than to chase.
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The International Alliance of ALS/MND Associations lists five approved medications, and which of them you can be prescribed depends on where you live:
- Riluzole
- Edaravone (Radicava)
- Tofersen (Qalsody) for SOD1 ALS
- Methylcobalamin (Rozebalamin) approved in Japan
- Nuedexta for pseudobulbar affect, not disease progression
These medications do not all serve the same purpose. Some aim to slow disease progression, while others target a specific genetic subtype or help manage symptoms.
Explained: what this word meansEvidence for this answer
The sources the Compass team used to write this answer. Highlighted phrases in the answer above correspond to the statements below.
Statement 1 of 1. The International Alliance's approved drugs page lists five medications, and which of them is available depends on the country.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
The Alliance's approved drugs page links to exactly five pages and no others: Rozebalamin, Riluzole, Radicava, Nuedexta and Tofersen. It carries no prose at all, so it shows what this organisation lists rather than a worldwide count of approvals, which is why the answer names the Alliance instead of asserting a global total. Each linked page names the countries that have approved that drug.
Link checked August 2026
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Riluzole is one of the most widely used medications for ALS/MND and is often offered soon after diagnosis.
It is thought to reduce damage to motor neurons by lowering levels of glutamate, a chemical that can be toxic in excess.
Clinical trials showed that riluzole can extend survival or delay the need for ventilation by around 2 to 3 months.
Some studies of people taking riluzole in everyday care have reported longer survival than that. In a 2020 review, eight of fifteen such studies found survival a median of 6 to 19 months longer. These studies were not randomised, so they cannot show that riluzole caused the difference, and the randomised trial evidence still points to around two to three months.
It is widely approved globally, including in Australia, the US, the UK, and across Europe, and is commonly used as a baseline treatment.
Explained: what this word meansEvidence for this answer
The sources the Compass team used to write this answer. Highlighted phrases in the answer above correspond to the statements below.
Statement 1 of 2. Riluzole extends survival, or delays the point at which a tracheostomy or ventilation is needed, by roughly two to three months on average.
Supports this. Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND) — Cochrane · Research review · June 2021
A systematic review pooling four randomised trials in 1477 people. It set out to measure exactly this: survival, and the delay before tracheostomy or mechanical ventilation. It found median survival rose from 11.8 to 14.8 months, and describes the effect as very modest.
“Riluzole 100 mg daily is reasonably safe and probably prolongs median survival by about two to three months in patients with amyotrophic lateral sclerosis.”
Authors' conclusionsLink checked August 2026
Statement 2 of 2. Some non-randomised studies of riluzole in routine care have reported longer survival than the trials did, and they cannot show that riluzole caused the difference.
Qualifies this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Reports much larger real-world figures than the trial estimate, from observational studies rather than randomised trials. A 2020 review found eight of fifteen such studies reported survival a median of six to nineteen months longer, so fewer than half found that gain. Nobody was randomised, so the difference cannot be attributed to the drug. Summarised because those sentences carry errors.
Link checked August 2026
Used across the whole answer
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Edaravone (Radicava) is a medication designed to reduce oxidative stress, which may help protect motor neurons from further damage.
Clinical trials showed a slowing of functional decline in a specific group of early-stage patients, with a difference of about 2.49 points on the ALSFRS-R over 24 weeks.
Its benefit appears strongest in people who start treatment earlier and meet fairly specific clinical criteria, so it is not considered equally effective for everyone with ALS/MND.
The wider evidence is mixed. Some studies suggest a small benefit and others show no benefit at all. A European trial called ADORE tested a different oral version of edaravone, made by another company, and it did not do better than placebo. It is unknown how comparable that version is to the approved edaravone, and nothing beyond a brief statement has been published comparing them.
It is approved in countries including the US, Japan, Australia, and Canada, but is not universally available or funded in all healthcare systems.
Evidence for this answer
The sources the Compass team used to write this answer. Highlighted phrases in the answer above correspond to the statements below.
Statement 1 of 5. The trial that showed a benefit for edaravone enrolled only people who met early-stage criteria, and found a 2.49 point difference on the ALSFRS-R over 24 weeks.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Gives the same figure, 2.49 ALSFRS-R points over 24 weeks, and the context behind it. The first trial found no significant benefit. Looking back at its results suggested people earlier in the disease had done better, and the second trial was then built around only those people, which is where the figure comes from.
“Follow up (post-hoc) analysis revealed that a subgroup of participants earlier in the disease appeared to benefit compared to those further progressed.”
Edaravone IV in Clinical TrialsLink checked August 2026
Statement 2 of 5. Edaravone's benefit looks strongest in people who start early and fit specific criteria, and it is not regarded as equally effective for everyone.
Qualifies this. International Alliance — International Alliance of ALS/MND Associations · Organisation
The Alliance's current assessment is more uncertain than the answer. It says the picture is still changing and that studies disagree, with some finding no benefit at all rather than a smaller one. A separate European trial, ADORE, tested a different oral edaravone made by another company and did not show a benefit over placebo.
“There are conflicting results with some studies suggesting a small benefit while other studies show no benefit.”
Current StatusLink checked August 2026
Statement 3 of 5. Studies of edaravone disagree. Some find a small benefit and some find none.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
The Alliance's current assessment says the picture is still changing and that studies disagree, with some finding no benefit at all rather than a smaller one.
“There are conflicting results with some studies suggesting a small benefit while other studies show no benefit.”
Current StatusLink checked August 2026
Statement 4 of 5. The ADORE trial tested TW001, an oral edaravone made by a different company from the approved product, and it did not do better than placebo.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Names the drug ADORE tested and who made it: TW001, also called FAB122, developed by Treeway in the Netherlands. This is not the approved oral edaravone, Radicava ORS, which comes from a different manufacturer and is covered separately on the same page.
“TW001, also called FAB122, was tested in a double-blind, placebo-controlled Phase 3 clinical trial across Europe, called the ADORE study, that did not demonstrate benefit versus placebo.”
Edaravone IV in Clinical TrialsLink checked August 2026
Statement 5 of 5. It is not known how comparable the edaravone tested in ADORE is to the approved product, and no detailed comparison has been published.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
States the comparability gap directly, in the same paragraph that describes the ADORE result. This is why the ADORE finding cannot be read across to the approved oral edaravone.
“It is unknown how comparable this form of edaravone is to the MT Pharma drug and no detailed comparison has been published to date beyond a brief statement in this publication.”
Edaravone IV in Clinical TrialsLink checked August 2026
Background the team read
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Tofersen (Qalsody) is a gene-targeted treatment designed specifically for people with SOD1-related ALS, which is a small genetic subtype of the disease.
It works by reducing the production of the toxic SOD1 protein that contributes to nerve cell damage.
While the early trial did not meet its main endpoint at 28 weeks, it showed promising biomarker changes and longer-term functional trends, particularly when treatment is started earlier.
Because it only applies to SOD1 ALS, genetic testing is important to determine whether this treatment is relevant for you.
Tofersen is given through intrathecal injection into the spinal fluid, so treatment involves lumbar punctures rather than tablets or standard infusions. It can only be given in a healthcare facility equipped for it, because delivery needs highly specialised staff and close monitoring. The dose is given every two weeks for the first three doses, then once every four weeks after that.
Reported side effects include pain in the arm, back, legs, muscle or joints. More serious reported side effects include inflammation of the spinal cord or irritation of the nerve roots, swelling of the optic nerve with raised pressure inside the skull, and inflammation of the linings of the brain. Ask your team to go through these with you before you start.
There are also new clinical trials planned and underway exploring whether similar approaches may be effective beyond the SOD1 genetic subtype, which could help shape future treatments for broader groups of people living with ALS.
It is currently approved in the US and some other regions, with access in some countries through early access or special programs.
Explained: what this word meansEvidence for this answer
The sources the Compass team used to write this answer. Highlighted phrases in the answer above correspond to the statements below.
Statement 1 of 6. Tofersen is only for people whose ALS is caused by a SOD1 gene fault, which is a small share of everyone with the condition.
Supports this. Your ALS Guide — Your ALS Guide · Practical guide
Puts a number on 'small': about 2 in every 100 people with ALS have a SOD1 mutation. It also notes that the mutation is found by genetic testing, which is why the answer says testing matters here.
“Tofersen can slow the rate of disease progression for the approximately 2% of ALS patients—those who have an SOD1 gene mutation (which can be detected through genetic testing).”
Qalsody® (tofersen)Link checked August 2026
Statement 2 of 6. Beyond its primary endpoint, tofersen lowered a marker of nerve damage and the longer follow-up favoured people who started treatment sooner.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Describes both parts. Lowering SOD1 produced a significant fall in neurofilament light chain, a marker of nerve damage, and secondary measures of movement, breathing, strength and quality of life pointed the right way. At twelve months the group treated from the start was doing better than the group that started six months later.
“Based on the available evidence, it is the opinion of the SAC that tofersen is likely to have a significant benefit for people living with ALS/MND caused by a mutation in the SOD1 gene, particularly in those starting treatment earlier.”
SummaryLink checked August 2026
Statement 3 of 6. Tofersen can only be given in a healthcare facility equipped for intrathecal administration, because delivery needs highly specialised personnel and close monitoring.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
States the setting requirement and the reason for it. This is why tofersen is not something that can be arranged locally like a tablet or a routine infusion.
“Because its delivery requires highly specialized personnel and close monitoring, it can only be delivered in a healthcare facility equipped for intrathecal administration.”
Dose and AdministrationLink checked August 2026
Statement 4 of 6. Tofersen is given every two weeks for the first three doses and then as a maintenance dose once every four weeks.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Gives the schedule. The four weekly dose is a maintenance dose, so the lumbar punctures continue for as long as someone stays on treatment rather than stopping after a course.
“The treated individual will receive a dose every two weeks for three times and then a maintenance dose once every four weeks.”
Dose and AdministrationLink checked August 2026
Statement 5 of 6. The most common reported side effects of tofersen include pain in the arm, back, legs, muscle or joints.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Lists this first among the most common side effects. The same list also records raised proteins or blood cells in the cerebrospinal fluid, and fever.
“Pain in the arm, back, legs, muscle or joints”
Reported Side EffectsLink checked August 2026
Statement 6 of 6. More serious reported side effects of tofersen include myelitis or radiculitis, papilledema with raised intracranial pressure, and aseptic meningitis.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Lists three more serious side effects under this heading: inflammation of the spinal cord or irritation of the nerve roots, swelling of the optic nerve with raised pressure inside the skull, and inflammation of the brain linings. The quote is the first of the three; the answer carries all three because the page presents them as one set.
“Inflammation of the spinal cord (myelitis) and/or irritation of the nerve roots (radiculitis)”
Reported Side EffectsLink checked August 2026
Background the team read
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Methylcobalamin (Rozebalamin) is a very high dose form of vitamin B12 that was approved in Japan in 2024 for ALS/MND.
An earlier trial of 373 people, run over 182 weeks, did not meet its main endpoints. A later trial, JETALS, enrolled 130 people in Japan who were within a year of symptom onset and had been declining at a moderate rate, defined as a fall of 1 to 2 points on the ALSFRS-R over a 12 week observation period beforehand, and 90% of them were already taking riluzole. Over 16 weeks, the methylcobalamin group differed from placebo by 1.97 points on the ALSFRS-R.
Measures of muscle strength and breathing showed no significant difference from placebo, the trial ran only 16 weeks, and because methylcobalamin reddens urine, participants may have been able to work out which group they were in. If you are further from your first symptoms than a year, or progressing faster or more slowly than that, you sit outside the group this trial studied.
It is not widely approved globally and is currently approved in Japan under the brand name Rozebalamin. Outside Japan, some people explore access through alternative legal pathways, but availability varies by country and should be discussed carefully with a treating clinician.
Some people living with ALS/MND also take other forms of high-dose vitamin B12, such as methylcobalamin sublingual tablets. Over-the-counter vitamin B12 has not been shown to work for ALS/MND. The dose used in the trials, and the dose approved in Japan, is 50 mg injected into a muscle twice a week.
There is also a safety point to weigh. The quality of over-the-counter supplements can be inconsistent, and high doses may be harmful. It is worth talking to your care team before starting a high-dose B12 supplement.
Evidence for this answer
The sources the Compass team used to write this answer. Highlighted phrases in the answer above correspond to the statements below.
Statement 1 of 6. An ultra-high dose form of methylcobalamin was approved for ALS/MND in Japan in 2024.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Gives the approval and its date, and notes that the dose approved for ALS/MND is far higher than the low-dose methylcobalamin already used in Japan for other nerve conditions.
“Based on the outcomes of a Phase 3 clinical trial, an ultra-high dose of methylcobalamin (50 mg) was approved in Japan on September 24, 2024, for the treatment of ALS/MND.”
BackgroundLink checked August 2026
Statement 2 of 6. The larger, longer trial that came before JETALS did not meet its main endpoints.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Describes this trial and its result. It ran in 44 Japanese centres in 373 people within three years of symptom onset, testing 25 mg and 50 mg twice weekly against placebo for 182 weeks, with survival and the ALSFRS as primary outcomes. Neither dose met them. A later look suggested a difference in those who joined within a year of onset, and JETALS was built to test that group.
“Both concentrations of methylcobalamin were found to be safe and well tolerated however, the primary endpoints were not met in either group.”
Clinical TrialsLink checked August 2026
Statement 3 of 6. JETALS enrolled 130 people in Japan within a year of symptom onset and declining at a defined moderate rate, most already on riluzole, and over 16 weeks the methylcobalamin group differed from placebo by 1.97 points on the ALSFRS-R.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Gives the figure and the entry criteria the answer now carries: 130 people across 25 Japanese centres, within a year of symptom onset, with progression defined as a 1 to 2 point fall in ALSFRS-R over a 12 week observation period beforehand, and 90% already taking riluzole. Summarised because the sentence carrying the number is grammatically incomplete on the page.
Link checked August 2026
Statement 4 of 6. In JETALS, muscle strength and breathing measures showed no significant difference from placebo, the trial lasted only 16 weeks, and participants may have been able to tell which group they were in.
Qualifies this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Lists all three limits itself. Muscle strength, forced vital capacity and Norris scale scores showed no significant difference from placebo, and the page calls the trial shorter than most ALS/MND trials at 16 weeks and notes that methylcobalamin's effect on urine colour may have let participants work out which group they were in.
“Additionally, muscle strength, forced vital capacity and Norris scale scores did not show significant differences compared to the placebo group.”
Clinical TrialsLink checked August 2026
Statement 5 of 6. Over-the-counter vitamin B12 has not been shown to be effective for ALS/MND, and the trials and the Japanese approval use 50 mg injected into a muscle twice a week.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Says the efficacy of over-the-counter oral vitamin B12 for ALS/MND is unproven, which is a statement that nothing has shown it works rather than a question of how well it works compared with injections. The same page gives the approved Japanese dose as 50 mg twice a week injected intramuscularly, and both trials used twice weekly intramuscular injections at 25 mg or 50 mg.
“While oral formulations of vitamin B12 are easily accessible over-the-counter, their efficacy for ALS/MND remains unproven.”
Current StatusLink checked August 2026
Statement 6 of 6. Over-the-counter supplement quality varies, and high doses of vitamin B12 may be harmful.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Raises this directly alongside the efficacy point, citing an ALSUntangled review from 2015. The Alliance does not say what dose becomes harmful or what the harm is, so the answer states the caution without putting a number on it.
“Furthermore, the quality of over-the-counter supplements can be inconsistent, and high doses may be detrimental to health”
Current StatusLink checked August 2026
Background the team read
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Nuedexta (dextromethorphan/quinidine) is used to treat pseudobulbar affect, a condition that causes sudden, uncontrollable episodes of laughing or crying.
It does not slow ALS/MND progression or help people live longer. In the trials that led to its approval, people taking Nuedexta rated their quality of life and their relationships better than people in the comparison groups.
A randomised, double-blind crossover trial in 60 people, published in 2017, tested whether Nuedexta improves speech and swallowing and reduces drooling, with each treatment period lasting 28 days. In that trial people rated their own bulbar function as better while taking it, and a preliminary result from a separate trial showed an improvement in bulbar physiology, which is a measurement rather than a rating. An independent ALSUntangled review supports considering Nuedexta for bulbar problems, with or without pseudobulbar affect. More research is still warranted.
This is off-label use. Nuedexta is approved only for pseudobulbar affect, so using it for speech and swallowing is not a licensed use anywhere. Its longer term effect on speech and swallowing is unclear, and one open label study found no benefit at one year.
Nuedexta also has safety considerations that go well beyond one heart rhythm problem, so it is not a medicine to weigh up on your own. The manufacturer's safety information asks people to tell their doctor before starting it about a range of things, including monoamine oxidase inhibitors (MAOIs) and quinidine or quinidine-related medicines, which cannot be combined with it, with MAOIs needing a 14 day gap either side; any previous allergic reaction to dextromethorphan or quinidine-like medicines; and any heart disease or family history of heart rhythm problems, since it can affect heart rhythm and your doctor may check yours before you start. Go through everything you take, including over-the-counter medicines and supplements, with your prescriber or pharmacist.
Nuedexta is approved for pseudobulbar affect in the United States only. It was approved in Europe after the US approval in 2010, but that approval was later withdrawn for commercial reasons. If you live outside the United States, it is worth asking your team whether it can be prescribed where you are.
Explained: what this word meansEvidence for this answer
The sources the Compass team used to write this answer. Highlighted phrases in the answer above correspond to the statements below.
Statement 1 of 8. Nuedexta treats pseudobulbar affect. It does not slow the progression of ALS/MND or help people live longer.
Supports this. Your ALS Guide — Your ALS Guide · Practical guide
States it directly, and separates the two things Nuedexta is and is not for: it does not change the course of the disease, but it helps with the sudden episodes of laughing or crying that pseudobulbar affect causes.
“Nuedexta® does not slow functional decline or help people live longer, but it can help regulate emotional control for ALS patients who experience pseudobulbar affect (PBA), which is characterized by sudden and unpredictable episodes of laughing or crying.”
Nuedexta®Link checked August 2026
Qualifies this. ALS Untangled — ALS Untangled · Research review
An independent graded review, last checked in June 2026, which puts this slightly differently. It reports an absence of evidence that Nuedexta slows progression, rather than evidence that it does not, which is a weaker claim than the answer makes.
“There is no evidence suggesting Nuedexta slows down progression or prolongs survival.”
Key InformationLink checked August 2026
Statement 2 of 8. In the approval trials, people taking Nuedexta rated their quality of life and their relationships higher than the comparison groups did.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Describes the first pivotal trial, in which people taking Nuedexta scored lower on the pseudobulbar affect symptom scale than people taking either component alone, and also rated their quality of life and relationships higher. The page does not quantify the size of that difference.
“Participants taking Nuedexta also rated their quality of life and quality of relationships higher than those in the other two groups.”
Study 1:Link checked August 2026
Statement 3 of 8. In a 2017 randomised crossover trial in 60 people, with 28 day periods, people rated their own bulbar function better on Nuedexta. A preliminary result from another trial showed improved bulbar physiology. An independent review supports considering it for bulbar problems and wants more research.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Describes the trial in detail: 60 people with ALS/MND, randomised, double-blind and crossover, 28 days on Nuedexta or placebo, a two week break, then the groups swapped. It records that ALSUntangled reviewed the same use and supports considering Nuedexta for bulbar dysfunction with or without pseudobulbar affect, and it adds that more research is warranted.
“In 2017, researchers recruited 60 people with ALS/MND for a randomized, double-blind, cross-over clinical trial looking at if the drug could improve speech and swallowing and reduce drooling.”
Current StatusLink checked August 2026
Supports this. ALS Untangled — ALS Untangled · Research review
An independent graded review, last checked June 2026, which reaches the same conclusion from the same trial and grades the trial evidence C. It notes that a preliminary result from another trial showed improvement in bulbar physiology.
“A well designed phase II trial in PALS demonstrated the efficacy of Nuedexta in patient-reported bulbar function, and the preliminary result of another trial showed improvement in bulbar physiology.”
Key InformationLink checked August 2026
Statement 4 of 8. Using Nuedexta for speech and swallowing is off-label, because its only approval is for pseudobulbar affect.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
Says the approval covers pseudobulbar affect only, and only in the United States, in the same section that describes the speech and swallowing trial. It describes what ALSUntangled reviewed as off-label use of Nuedexta for improving bulbar function. That sentence is not quoted here because it carries a spelling error on the page.
Link checked August 2026
Statement 5 of 8. Nuedexta's longer term effect on speech and swallowing is unclear, and one open label study found no benefit at one year.
Qualifies this. ALS Untangled — ALS Untangled · Research review
Sets this caution alongside its support for trying Nuedexta for bulbar problems: the long term effect on bulbar function is unclear and one open label study found no benefit at one year. It grades the risks C and suggests checking periodically whether it is still helping.
“However, its long term effect on bulbar function is unclear, and one open label study showed a lack of benefit in one year.”
Key InformationLink checked August 2026
Statement 6 of 8. Nuedexta cannot be combined with MAOIs or with quinidine and quinidine-related medicines, and MAOIs need a 14 day gap either side.
Supports this. Nuedexta official website — Nuedexta · Official clinical information
The manufacturer's own safety information, which lists this first among the things to tell a doctor before starting Nuedexta and states the 14 day separation for MAOIs explicitly. The same section also covers allergic reactions to quinidine-like medicines, heart rhythm problems and signs of serotonin syndrome, which is why the answer routes people to their prescriber rather than listing it all.
“If you are taking monoamine oxidase inhibitors (MAOIs), quinidine, or quinidine-related drugs. These can interact with NUEDEXTA causing serious side effects. MAOIs cannot be taken within 14 days before or after taking NUEDEXTA.”
IMPORTANT SAFETY INFORMATION:Link checked August 2026
Statement 7 of 8. Nuedexta can affect heart rhythm, so a doctor may check heart rhythm before someone starts it, and heart disease or a family history of rhythm problems needs raising first.
Supports this. Nuedexta official website — Nuedexta · Official clinical information
States that Nuedexta may cause serious side effects including changes in heart rhythm, that it may not be right for people with certain heart problems, and that a doctor may test heart rhythm before starting it.
“If you have had heart disease or have a family history of heart rhythm problems. NUEDEXTA may cause serious side effects, including changes in heart rhythm.”
IMPORTANT SAFETY INFORMATION:Link checked August 2026
Statement 8 of 8. Nuedexta's approval for pseudobulbar affect covers the United States only.
Supports this. International Alliance — International Alliance of ALS/MND Associations · Organisation
States the approval position directly. The page also records that Nuedexta was approved by the European Medicine Agency after its 2010 US approval and that the approval was later withdrawn for commercial reasons, and its country table lists the USA alone.
“While clinical trials have shown promising results, Nuedexta is still only approved for the treatment of PBA, and only in the United States.”
Current StatusLink checked August 2026
Used across the whole answer
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No currently approved treatments stop or reverse ALS/MND.
In randomised trials, riluzole extended median survival by about two to three months. Treatments may also slow functional decline in certain groups, or improve specific symptoms.
In practice, most people use a combination of approaches, including medications, supportive care, and assistive technologies, depending on their goals and stage of disease.
Evidence for this answer
The sources the Compass team used to write this answer. Highlighted phrases in the answer above correspond to the statements below.
Statement 1 of 2. None of the approved treatments halts or reverses ALS/MND.
Supports this. Your ALS Guide — Your ALS Guide · Practical guide
Says the same thing at the top of its rundown of the approved drugs, and says it without softening: they can slow decline modestly and add time, and they do not stop or reverse the disease. Its list covers the drugs approved in the United States, so it does not speak to methylcobalamin, which is approved only in Japan.
“These drugs can modestly slow functional decline and help people live longer, though they do not stop or reverse ALS.”
Approved Drugs for ALSLink checked August 2026
Statement 2 of 2. In randomised trials riluzole extended median survival by about two to three months.
Supports this. Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND) — Cochrane · Research review · June 2021
Pools four randomised trials of riluzole and puts this size on the survival gain. The review calls the beneficial effects very modest. It covers riluzole only, which is why the answer names riluzole rather than approved treatments as a group.
“Riluzole 100 mg daily is reasonably safe and probably prolongs median survival by about two to three months in patients with amyotrophic lateral sclerosis.”
Authors' conclusionsLink checked August 2026
Supports this. Your ALS Guide — Your ALS Guide · Practical guide
Gives the same figure for riluzole from the original studies, as an average of three months. It adds that the increase may be longer, which comes from studies of routine care rather than from randomised trials, so the answer keeps the randomised figure.
“Initial studies showed that riluzole helped people live an average of three months more, though increased survival time may be longer.”
Rilutek® (riluzole)Link checked August 2026
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Clinical trials are one of the main ways new ALS/MND treatments are tested.
You can search for trials using registries like ClinicalTrials.gov, along with other regional databases.
To take part in a trial, you will usually need to meet specific eligibility criteria, such as:
- stage of disease
- genetic subtype
- respiratory function
- prior treatments
Your neurologist or care team can help you understand whether a trial is a good fit and assist with referrals where needed.
Access to trials can also depend on location and study availability, so it is worth asking early and revisiting options over time.
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Not all ALS/MND treatments are approved or available in every country.
This is because each country has its own regulatory process for assessing safety and effectiveness. These decisions are based on:
- clinical trial evidence
- cost and funding considerations
- local healthcare system priorities
As a result, a treatment may be:
- approved in one country but not another
- available only through early access or special programs
- accessible privately but not funded through public systems
In some cases, people explore access outside their home country or through alternative pathways, but this should be done carefully and ideally with medical guidance.
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There is a wide range of experimental and alternative treatments discussed in ALS/MND, and not all are supported by evidence.
A more legitimate treatment will usually have:
- published clinical research, ideally in peer reviewed journals
- clear information about how it works and what was tested
- transparency around risks, side effects, and limitations
Be especially cautious of treatments that:
- promise a cure or guaranteed results
- rely heavily on testimonials rather than data
- require large upfront payments without clear evidence
- use vague language about being natural, detoxifying, or breakthrough
- heavily promote areas like natural herbs or stem cell treatments without strong ALS specific evidence
Where possible, discuss any treatment you are considering with your care team so you can weigh potential benefits against risks.
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There is rarely a single “right” treatment plan for ALS/MND. Most people weigh a mix of approved medications, symptom management, clinical trials, supplements, devices, and experimental options.
A good place to start is usually with approved treatments, because they have been tested in clinical trials, have known safety profiles, and offer measurable benefits for some people.
Many people living with ALS also experiment with supplements, devices, and other approaches. Some of these may show early signals of helping, but it is important to consider the individual risk profile, how strong the evidence is, and how they may interact with approved medications or other treatments you are already using.
When considering any treatment, it helps to ask:
- What is the best evidence behind it?
- What are the side effects or treatment burden?
- Will it help with survival, function, speech, swallowing, or quality of life?
- Is the cost financial, physical, or emotional?
- Does it fit my personal goals right now?
Some treatments require regular blood tests, infusions, lumbar punctures, PEG-compatible delivery, or significant ongoing monitoring. Others may be easy to access but have little real evidence behind them.
Be especially cautious of expensive private treatments, stem cell clinics, or therapies that promise a cure without strong published research.
Many people start with approved treatments as a baseline, then explore clinical trials or additional approaches alongside this.
Your priorities may also change over time. What feels right early after diagnosis may look different later, and it is okay to reassess.
The best decisions are usually made with both good information and honest conversations with your care team.
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