Cohort overview
80Patients in motor neuron disease cohort 0Deaths observed 2Medications meeting min-5 threshold 1ATC classes representedALS clinical-interest medications · always shown, regardless of min-exposed threshold · k-anonymized at k=2
The five medications below have particular relevance to ALS clinical care and research: the three FDA-approved disease-modifying therapies, the FDA-approved-for-PBA combination commonly used off-label in ALS care, and the briefly-approved combination withdrawn after confirmatory trial failure. Presence and year-range are reported regardless of whether the medication meets the standard min-exposed threshold; counts are k-anonymized (values below k= 2 reported as “< 2”).
| Medication | FDA status | Cohort presence | Unique patients | Year range | Records |
|---|---|---|---|---|---|
| Riluzole Rilutek, Tiglutik, Exservan · ATC N07XX02 | FDA-approved FDA-approved for ALS (1995) | Not observed | 0 | — | 0 |
| Edaravone Radicava, Radicava ORS · ATC N07XX14 | FDA-approved FDA-approved for ALS (2017) | Not observed | 0 | — | 0 |
| Tofersen Qalsody · ATC N07XX17 | FDA-approved FDA-approved for SOD1 ALS (2023, accelerated) | Not observed | 0 | — | 0 |
| Dextromethorphan-quinidine Nuedexta · ATC N07XX59 | Off-label-of-interest FDA-approved for PBA; off-label-of-interest in ALS | Not observed | 0 | — | 0 |
| Sodium phenylbutyrate-taurursodiol Relyvrio, Albrioza · ATC — | Withdrawn FDA-approved for ALS 2022; withdrawn 2024 after PHOENIX | Not observed | 0 | — | 0 |
Medication summary · grouped by ATC class · k=≥2 for "n_exposed"
Other nervous system drugs (motor neuron disease)
| Medication | n exposed | n unexposed | deaths (exp) | deaths (unexp) | median surv (exp) | median surv (unexp) | direction |
|---|---|---|---|---|---|---|---|
| edaravone N07XX14 | 6 | 74 | 0 | 0 | 15.4y | 9.9y | ↑ exposed longer |
| riluzole N07XX02 | 65 | 15 | 0 | 0 | 9.9y | 9.9y |
Methodology and limitations
This module produces the cohort assembly and exposure assignment described in Reimer et al. Lancet Digital Health 2026. It does not run the propensity score matching or the Cox proportional hazards analysis; those steps require dedicated tools (lifelines, statsmodels) and per-cohort tuning of caliper widths, immortal-time bias correction, and covariate balance assessment. The cohort dataset emitted by this module (drug_repurposing_cohort.csv) is the input to those tools.
Limitations of this analysis at any stage: small-cohort statistical power, inadequate covariate adjustment for known and unknown confounders, indication bias (a patient prescribed a PDE5 inhibitor may by virtue of that indication be healthier than average), immortal-time bias if exposure timing is mishandled, accuracy of EHR-coded motor-neuron-disease diagnosis, and lack of dose-response information in the bundle. Treat outputs as hypothesis-generating; prospective randomized trials remain the gold standard.
Cure ID intake export: produces one row per exposed (patient, medication) for submission to the FDA / NCATS-NIH / Critical Path Institute CURE Drug Repurposing Collaboratory Treatment Registry. Banded fields and pseudonymized identifiers comply with the registry's PII-free expectations.