An oral treatment with randomized evidence of a modest survival benefit in ALS.
What the evidence says
The pivotal trial studied 959 people. A Cochrane synthesis estimated roughly two to three months of median survival benefit in the trial populations; that average is not a prediction for an individual.
Keep in perspective
It does not regenerate motor neurons. Treatment choice, formulation and monitoring belong in a discussion with the treating neurologist.
Sources, India & access2 sources
For readers in India
Ask the prescribing team and a licensed pharmacy about the appropriate formulation, current supply and a written price quotation.
Edaravone is approved in the US for ALS. The oral suspension provides an alternative to intravenous administration and can also be given through a feeding tube.
What the evidence says
FDA describes evidence of less decline in daily functioning in a six-month trial. Oral approval used comparable drug exposure to the IV product. This supports slowing decline in the studied population, not recovery of lost function.
Keep in perspective
Limited evidence in a particular disease stage does not prove that every person outside the trial criteria cannot benefit. Avoid blanket exclusions based on wheelchair or ventilator use.
Sources, India & access1 source
For readers in India
Verify the exact product and route locally. US approval of Radicava ORS does not establish its routine availability in India.
Approved for adults with ALS and a SOD1 mutation. The FDA decision was based on lowering blood neurofilament light (NfL), a marker of nerve injury.
What the evidence says
The 28-week VALOR trial did not establish a significant primary clinical benefit. Longer follow-up associated earlier treatment with less decline, but the open-label comparison has limitations. Clinical benefit remains under confirmatory study.
Keep in perspective
A biomarker response is not proof of a cure or predictable recovery. Intrathecal treatment requires a specialist team. Investigation in non-SOD1 ALS does not expand the approved indication.
Sources, India & access2 sources
For readers in India
Confirm the genetic result with a specialist. Local authorization, supply, import permission and funding must be checked for the individual patient.
Japan approved this injectable methylcobalamin formulation for slowing functional impairment in ALS. It is distinct from routine vitamin B12 supplementation.
What the evidence says
JETALS found less ALSFRS-R decline over 16 weeks in a selected early-stage Japanese population. The PMDA review supports the indication while documenting the studied population and safety requirements.
Keep in perspective
Do not generalize the short trial to all stages or substitute ordinary supplements. Rozebalamin is mecobalamin (E0302), not MT-1186.
Sources, India & access3 sources
For readers in India
Japanese authorization does not mean approval or reliable supply in India. Discuss product identity and any lawful access route with the clinical team.
A new Phase 1/2 study evaluates a medicine intended to restore UNC13A function in ALS.
What the evidence says
The registry was updated on 9 September 2026. It describes a randomized, blinded, placebo-controlled trial assessing safety, tolerability and early signals after intrathecal administration.
Keep in perspective
No results are posted. A newly recruiting trial is an opportunity to test a hypothesis, not evidence of clinical benefit.
Sources, India & access1 source
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
The Phase 3 PREVAiLS study tests pridopidine in people with early, rapidly progressive ALS.
What the evidence says
First enrollment was announced on 30 March 2026; the registry still lists recruiting. The earlier HEALEY trial did not meet its primary endpoint. Subgroup observations motivated a more focused confirmatory trial.
Keep in perspective
Subgroup findings are hypothesis-generating. PREVAiLS has not yet established efficacy or supported ALS marketing approval.
Sources, India & access3 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
An investigational therapy targeting cellular energy pathways; CNM-Au8 remains under development for ALS.
What the evidence says
Clene’s 14 August update targets an accelerated-approval application in early Q4 2026. Its HEALEY trial did not show significant benefit on the primary 24-week progression endpoint. Later biomarker and survival analyses underpin the planned submission.
Keep in perspective
A planned application, FDA meeting or exploratory analysis is not approval. RESTORE-ALS should not be described as enrolling without a verified launch.
Sources, India & access2 sources
For readers in India
No routine Indian access or local trial site is established by these sources. Expanded access is distinct from marketing approval.
ENDURANCE is the planned confirmatory study of cells prepared from a participant’s own bone marrow.
What the evidence says
The August 2026 sponsor update describes preparations to open the study; the registry lists not yet recruiting. An FDA-agreed Special Protocol Assessment concerns trial design and does not establish that the treatment works.
Keep in perspective
The new trial has no posted results. Do not present NurOwn as an approved stem-cell treatment or imply that protocol agreement guarantees approval.
Sources, India & access2 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
A confirmatory Phase 3 study is registered for this investigational kinase inhibitor.
What the evidence says
The registry lists not yet recruiting. In April 2026 AB Science described the ALS study as not yet started and a temporary European trial halt. EMA’s earlier refusal cited unreliable study data and insufficiently demonstrated benefit.
Keep in perspective
Post-hoc or preprint survival analyses do not overturn the regulatory refusal or establish an approved treatment.
Sources, India & access3 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
A Phase 3 trial in ALS associated with pathogenic FUS mutations.
What the evidence says
The July 2026 registry lists active follow-up with enrollment closed and no results posted. The study evaluates efficacy and safety of lowering FUS protein.
Keep in perspective
This genetically defined program cannot be generalized to all ALS. An anticipated readout is not a completed positive result.
Sources, India & access1 source
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
Belgium, Brazil, Canada, Germany, Ireland, Italy, Japan, Netherlands, Poland, South Korea, Spain, Sweden, Switzerland, Taiwan, United Kingdom, United States
An antisense approach intended to restore stathmin-2 expression affected by TDP-43 dysfunction.
What the evidence says
QurAlis reported interim target-engagement and biomarker findings in February 2026, with an overall trend and subgroup signals in function. The sponsor calls ANQUR Phase 1/2; the registry classifies it Phase 1 and lists enrollment closed.
Keep in perspective
Interim and subgroup results are not confirmatory efficacy. A proposed 2027 Phase 3 program remains a plan.
Sources, India & access2 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
An investigational fixed-dose formulation of ciprofloxacin and celecoxib.
What the evidence says
On 31 August 2026 NeuroSense announced plans to optimize PARAGON into a smaller, shorter pivotal study, subject to FDA alignment. A Canadian submission is targeted for December 2026. Neither milestone should be described as completed.
Keep in perspective
Earlier clinical and biomarker signals require confirmation. The formulation is not interchangeable with self-combining its component medicines.
Sources, India & access1 source
For readers in India
An Indian approval, launch date or price is not established by this development announcement.
A combination biologic strategy intended to enhance regulatory T-cell activity.
What the evidence says
The August update reports continuing enrollment and targets topline data in Q1 2027. The sponsor describes ALSTARS as Phase 2; ClinicalTrials.gov lists Phase 2/3. Both descriptions refer to the same study.
Keep in perspective
No randomized efficacy results are posted. Trial phase and Fast Track designation are not evidence of approval.
Sources, India & access2 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
An investigational oral medicine designed to support clearance of harmful cellular proteins.
What the evidence says
The HEALEY trial center reports enrollment complete, consistent with the July 2026 registry status. The Phase 2/3 cohort is now in follow-up; no efficacy results are posted.
Keep in perspective
Small earlier safety studies and open-label observations do not establish slowing of ALS progression.
Sources, India & access2 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
A small molecule being studied for effects on synaptic function; clinical benefit in ALS remains unconfirmed.
What the evidence says
The early study is registered as completed. The sponsor reported Phase 2a findings in November 2025. A separate US expanded-access protocol is listed as available (NCT07088159).
Keep in perspective
Early functional observations and access outside a trial do not prove synapse regeneration or reliable functional recovery in people with ALS.
Sources, India & access3 sources
For readers in India
The US expanded-access protocol is not an Indian approval or a guarantee that an overseas patient can join.
A therapeutic antibody program derived from research into biological resilience.
What the evidence says
Alchemab announced a first-in-human Phase 1 trial on 9 September 2025. The program was licensed to Lilly. No later ALS efficacy result was established in this review.
Keep in perspective
A licensing agreement does not validate efficacy. Current patient recruitment and a Phase 2 start date have not been verified.
Sources, India & access1 source
For readers in India
No Indian trial site or commercial access is verified. Corporate presence in India does not establish future access.
A Phase 2 study of an intravenous peptide approach to neuroprotection and blood–brain barrier function.
What the evidence says
June 2026 preliminary results cover 82 randomized participants. Axoltis reported exploratory functional and biomarker trends; the cited early NfL comparison was not statistically significant.
Keep in perspective
Preliminary conference findings need the full controlled analysis. Biomarker trends must not be presented as proven survival benefit.
Sources, India & access2 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
A Phase 1 ALS study in China evaluates induced-pluripotent-stem-cell-derived motor neuron progenitor cells.
What the evidence says
The ALS-specific record NCT07118319 lists recruiting and no posted results. Separate XS228 spinal-cord-injury trials must not be used as evidence of benefit in ALS.
Keep in perspective
Safety, integration of transplanted cells and clinical benefit remain experimental. There is no established restoration of lost motor neurons or function.
Sources, India & access1 source
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
A Phase 1 study of an antisense medicine targeting calpain-2.
What the evidence says
Amylyx’s June 2026 update reported no drug-related serious adverse events in the lowest-dose cohort, while the measured biomarkers remained near baseline. Higher-dose cohorts are being evaluated.
Keep in perspective
Tolerability at a low dose is not evidence of clinical efficacy. Dose-escalation findings are still early.
Sources, India & access2 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
SARM1 inhibition has now entered ALS patient testing with this oral investigational drug.
What the evidence says
Nura Bio announced the first ALS participant dosed in June 2026. The Phase 1b/2a program measures safety, drug exposure and biomarkers; registry sites are in Australia and Canada.
Keep in perspective
The biological rationale and healthy-volunteer findings do not yet demonstrate benefit in ALS. This program is distinct from preclinical ASHA-624.
Sources, India & access2 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
An early gene-therapy study intended to clear pathological TDP-43.
What the evidence says
Celosia announced first dosing in March 2026 at Macquarie University Hospital. The Phase 1b study is open-label and focuses on safety and tolerability. The recruiting registry record was last updated in February.
Keep in perspective
The registry is older than the dosing announcement: confirm current enrollment directly. Reversal in animal models is not evidence of reversal in people.
Sources, India & access2 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
A July 2026 Nature Medicine paper reports early human testing of an RNA-silencing approach for SOD1 ALS.
What the evidence says
The published first study involved six participants and examined safety, exposure and preliminary biological or clinical observations. A separate Phase 1 trial is registered as recruiting.
Keep in perspective
Six participants without a definitive controlled efficacy comparison cannot establish survival benefit or superiority to tofersen. Enrollment status needs confirmation because the cited registry update is older.
Sources, India & access2 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
A Phase 2b randomized study of an oral NLRP3 inflammasome inhibitor.
What the evidence says
The 1 September 2026 registry update lists recruiting, with multiple countries participating and no results posted. This is an investigational ALS program associated with an Indian-origin sponsor.
Keep in perspective
Sponsor origin does not mean a trial is taking place in India or that the medicine is approved there.
Sources, India & access1 source
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
A first-in-human Phase 1/2 program using a vectorized antibody approach directed at TDP-43 pathology.
What the evidence says
The registry updated on 10 September 2026 lists recruiting at sites in Europe and the US. It is an open-label ascending-dose study with clinical and biomarker assessments.
Keep in perspective
No results are posted. Small uncontrolled gene-therapy studies establish early safety information, not a proven ALS treatment. The older sponsor mechanism page still says preclinical; use the dated registry for current trial status.
Sources, India & access2 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
A single-dose Phase 1 study in adults with SOD1 ALS or ALS without a known ALS-related genetic mutation.
What the evidence says
The September 2026 registry lists recruiting in the US. The study evaluates safety, tolerability and pharmacodynamic effects after intrathecal administration.
Keep in perspective
There are no posted efficacy results. Eligibility is protocol-specific and must be confirmed by the study team.
Sources, India & access1 source
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
An exploratory trial tests whether tofersen has biological or clinical effects beyond genetically confirmed SOD1 ALS.
What the evidence says
The registry updated in June 2026 lists a US Phase 2 study and excludes SOD1- and FUS-associated disease. This is a research question with no posted results.
Keep in perspective
The FDA-approved indication remains SOD1 ALS. This trial is not evidence to prescribe tofersen broadly for sporadic ALS.
Sources, India & access1 source
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
DAZALS missed its primary functional endpoint. Corcept is pursuing further study after secondary and exploratory survival findings.
What the evidence says
The April 2026 sponsor report describes two-year survival analyses and a planned Phase 3 trial. The long-term comparisons include selected participants who did or did not enter the extension. The registry still says recruiting, which should not be read as enrollment in the planned pivotal study.
Keep in perspective
Survival signals after a failed primary endpoint need confirmation, especially with selection into an extension. No results posted in a registry does not mean results have not been reported elsewhere. Confirm any enrollment directly.
Sources, India & access2 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
A new Phase 1/2 study investigates an RNA-targeted approach to abnormal lipid metabolism in ALS.
What the evidence says
Leal announced first dosing in June 2026. The August registry lists a blinded, placebo-controlled dose-escalation study assessing safety, tolerability and pharmacological effects.
Keep in perspective
The rationale extends to sporadic and genetic ALS, but clinical benefit has not been established. This is a first-in-human program with no posted results.
Sources, India & access2 sources
For readers in India
No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.
ASHA-624 is a molecular-glue strategy designed to hold SARM1 inactive and protect axons.
What the evidence says
The identified funder announcement supports preclinical studies in an inherited optic-nerve disease. It does not establish an ALS clinical trial or human ALS efficacy.
Keep in perspective
Do not equate promising work in another disease model with an available ALS therapy. NB-4746 is the distinct SARM1 program now in ALS clinical testing.
Sources, India & access2 sources
For readers in India
Research only; no verified clinical access for ASHA-624.
Researchers are investigating precise removal of disease-associated repeat RNA while preserving normal gene activity.
What the evidence says
A 2025 Nature Communications study used a high-fidelity Cas13 system in experimental models of C9ORF72-linked disease and reduced molecular abnormalities.
Keep in perspective
This is laboratory and animal-model evidence. Delivery, off-target effects and long-term safety need evaluation before claims of human benefit.
Sources, India & access1 source
For readers in India
No approved CRISPR treatment for ALS is established by this study.
The medicine was removed from the US and Canadian markets after its confirmatory trial failed.
What the evidence says
Amylyx announced withdrawal in April 2024 following PHOENIX, which did not meet its prespecified primary and secondary endpoints.
Keep in perspective
Earlier approval and positive small-study results do not outweigh a failed confirmatory trial. Current care should be discussed with the treating clinician.
Sources, India & access1 source
For readers in India
Do not present this as a currently marketed ALS treatment or recommend purchasing a substitute combination.
The tested eIF2B activator did not establish efficacy in the HEALEY study.
What the evidence says
Calico reported in January 2025 that neither the primary nor exploratory higher dose met the primary endpoint. Key secondary endpoints for the primary dose also were not significantly different from placebo.
Keep in perspective
Exploratory findings cannot be presented as an approved benefit. A negative trial does not, by itself, prove every future study of the mechanism must fail.
DNL343 did not demonstrate slowing of ALS progression in the tested regimen.
What the evidence says
The January 2025 announcement reports that the primary combined function/survival endpoint and key strength and respiratory secondary endpoints were not met at 24 weeks.
Keep in perspective
Safety or a biological rationale cannot substitute for a demonstrated clinical effect.
The HIMALAYA Phase 2 program did not meet its ALS efficacy objective.
What the evidence says
The study registry records termination for lack of efficacy. This entry concerns the ALS study, not results from the separate multiple-sclerosis program.
Keep in perspective
Evidence from a different disease cannot be used to imply efficacy in ALS.
Dedicated eye-gaze and speech-generating products can provide communication and computer access as movement changes.
What the evidence says
Manufacturer documentation distinguishes a computer eye tracker from integrated Windows and iPad-based speech devices. Selection depends on positioning, vision, fatigue and the person’s preferred communication system.
Keep in perspective
Product documentation describes capabilities, not a guarantee of individual performance. Assess with an AAC professional and trial the exact setup.
Sources, India & access1 source
For readers in India
Request a local assessment and written quotation covering hardware, mount, software, warranty, repairs and language support.
A wearable, screen-free approach uses eye movements with audio feedback to communicate needs.
What the evidence says
An ALS feasibility study is registered, while current manufacturer documentation also describes a bedside hospital platform. Product configurations and clinical use cases must be distinguished.
Keep in perspective
A completed feasibility study does not guarantee reliable use for every person with advanced ALS. Verify the specific product and evidence with the supplier.
Sources, India & access2 sources
For readers in India
Current Indian distribution, home-use suitability and support have not been verified; contact the clinical team and manufacturer before purchase.
OptiKey supports typing, speech output and computer control using compatible input devices.
What the evidence says
The maintainers explicitly removed support for Tobii Eye Tracker 5, EyeX and 4C in OptiKey 4. Legacy OptiKey 3.2.x supports these older use cases.
Keep in perspective
Do not assume the latest OptiKey works with the Tobii Eye Tracker 5 setup elsewhere in this project. Verify hardware, driver, Windows and application versions together.
Sources, India & access1 source
For readers in India
Assess the existing hardware first. Budget for setup and assistance even where the software is free.
Neon provides gaze and other eye signals for researchers and developers; it is not a complete ALS communication system.
What the evidence says
The official real-time API can stream data to custom applications on the same network. That capability enables prototyping but does not establish clinical reliability for ALS.
Keep in perspective
Building a usable communication interface requires software, calibration/positioning work and individual testing. Do not equate raw gaze streaming with ready-to-use AAC.
Sources, India & access1 source
For readers in India
Consider established AAC assessment first. A custom integration needs sustained technical support and a backup communication method.
Head-tracking software can move a cursor using movements observed by a webcam.
What the evidence says
Enable Viacam’s documentation describes camera and tracking-area configuration. This is an access method for someone who can comfortably produce the required movements.
Keep in perspective
It is head tracking, not eye tracking. Weakness, fatigue and positioning can limit use; these tools are not established replacements for eye-gaze AAC.
Sources, India & access1 source
For readers in India
Trial comfort and accuracy with an assistive-technology professional. Maintain a simple communication backup.
Marker-based and smartphone experiments should be treated as engineering prototypes.
What the evidence says
No ALS-specific clinical validation or supported complete communication product was established for the older page’s proposed DIY setup. The Pupil Labs API documentation supports only the general feasibility of custom data integrations.
Keep in perspective
Do not present a prototype, price estimate or working demo as reliable daily communication for a person with ALS.
Sources, India & access1 source
For readers in India
Keep prototypes optional and supervised. They should not replace a dependable way to express urgent needs.
Research systems have enabled an individual with ALS to produce synthetic speech directly from recorded brain activity.
What the evidence says
UC Davis reported real-time voice synthesis in June 2025 in a participant with ALS. This is a communication milestone rather than restoration of natural speech muscles.
Keep in perspective
Small participant numbers, implanted hardware and intensive specialist support limit generalization. These systems are not established routine home products.
Sources, India & access1 source
For readers in India
Access is through formal research eligibility. Maintain practical AAC planning alongside interest in BCI research.
A 2026 early-feasibility study evaluates an implanted interface for communication and computer control.
What the evidence says
The trial registry lists recruiting in the US and includes people with severe speech or movement impairment. Safety and device feasibility are central objectives.
Keep in perspective
A research authorization is not commercial approval. Surgery, long follow-up and strict eligibility distinguish this from buying an eye tracker.
Sources, India & access1 source
For readers in India
No Indian study site is listed. Contact a formal research center about eligibility rather than relying on commercial availability claims.
India’s participation in a Target ALS-funded genome-wide association study broadens the ancestry represented in ALS genetics.
What the evidence says
The funder describes an India–UK collaboration involving NIMHANS, with genetic analysis and longitudinal samples. This is research into disease risk and biology, not a treatment trial.
Keep in perspective
Research participation is not a guarantee of free clinical genetic testing, a diagnosis or access to a targeted medicine.
Sources, India & access1 source
For readers in India
Ask the treating neurology center about genetic counseling, clinically appropriate testing and any currently open research protocol.
Following participants over time can connect molecular changes with clinical progression.
What the evidence says
The 2025 Global Natural History / Longitudinal Biofluid Study poster identifies AIIMS New Delhi and NIMHANS among participating institutions. Target ALS’s January 2026 update also describes tissue and genomic research resources.
Keep in perspective
Institutional involvement does not prove that a specific clinic is recruiting today or providing experimental treatment.
Sources, India & access2 sources
For readers in India
Confirm the exact protocol and recruitment status directly with the institution before arranging travel.
A treatment’s approval abroad, a company’s Indian presence and trial eligibility are separate questions.
What the evidence says
CDSCO provides a personal-use import process: Form 12A is the application and Form 12B is the permit. Permission, prescription, product supply and clinical suitability require separate confirmation.
Keep in perspective
Import permission is not Indian marketing approval or evidence that a medicine works. This page does not certify current prices, reimbursement or supply.
Sources, India & access1 source
For readers in India
Work with the treating team and licensed supply channels. Obtain written confirmation before committing to treatment-related travel or expense.
This review supports discussion with your ALS team. Individual suitability, genetic results and local access need clinical assessment. Research participation is voluntary, and a study listing does not guarantee eligibility.
A curated review of all previously included programs, with selected important additions. It is not an exhaustive registry of every ALS study worldwide.
01
Approval has a specific scope
Check the country, indication and patient population. Accelerated approval can depend on a biomarker while clinical benefit is still being confirmed.
02
A signal needs confirmation
Small studies, subgroup findings and open-label extensions can guide future trials. They carry different weight from a positive, controlled primary endpoint.
03
Plans can change
Enrollment, submissions and readouts are dated milestones. A sponsor’s planned next step is shown as a plan until there is evidence it happened.
Review method & limitations
Evidence cutoff: 10 September 2026. Sources were retrieved on 11 September 2026; dated developments after the cutoff are excluded.
Regulators establish approved indications. Trial registries establish the recorded design, phase and recruitment status. Registry dates are shown because records can lag behind events.
Published controlled results take precedence over subgroup claims. Sponsor announcements are labeled and used for operational milestones or explicitly preliminary findings.
Undated manufacturer pages describe product capabilities only; they do not establish a dated approval or verified availability on the cutoff date.
A biomarker measures a biological process. A change in NfL, TDP-43 or another marker is not automatically a demonstrated clinical benefit.
No medical specialist sign-off is claimed. Treatment suitability, genetic findings, access and current recruitment should be confirmed with the relevant clinical team.
What changed in this edition
Replaced unsupported stage-by-duration rules and blanket treatment recommendations with study-population and evidence limitations.
Separated Nuedexta symptom treatment from the four ALS-directed medicines reviewed here; clarified jurisdiction-specific approvals.
Corrected Rozebalamin to mecobalamin / E0302; removed the incorrect MT-1186 identification.
Corrected CNM-Au8, NurOwn, masitinib, PrimeC and NUZ-001 milestones using dated sources and registry records.
Added newer UNC13A, SARM1, RNA, gene-therapy and communication studies; kept every original topic or a clearly explained successor entry.
Made the previously hidden withdrawal, communication and experimental-technology content visible.
Removed unsupported Indian price, import-access and future availability promises; corrected Form 12A versus Form 12B.
Corrected OptiKey 4 versus legacy Tobii compatibility and removed unsourced device approval and superiority claims.
Research terms, in plain language
ALSFRS-R
A questionnaire used to track everyday functions such as speech, swallowing, movement and breathing.
Neurofilament light (NfL)
A marker associated with nerve injury. A lower level alone does not establish that someone functions better or lives longer.
Primary endpoint
The main outcome a trial is designed to test. Other or subgroup findings need careful interpretation when this outcome is not met.
Open-label extension
Follow-up in which participants and researchers know the treatment being given; comparisons are less protected from bias.
AAC
Augmentative and alternative communication: tools and strategies that support expression when speech is difficult.
Go to the original evidenceSource library78 references ↓
All sources retrieved 11 September 2026. Dates below are publication or registry-update dates; undated pages are identified. External links may change after this review.