ALS Treatments: Current Therapies & Emerging Research

FDA‑Approved Disease‑Modifying Drugs

• Riluzole (1995) – Glutamate release inhibitor; extends survival ~3–6 months. Oral 50 mg BID.
• Edaravone (2017 US) – Free‑radical scavenger; slows functional decline (ALSFRS‑R) in early‑stage. IV or oral formulation.
• Sodium phenylbutyrate‑taurursodiol (Relyvrio/Albrioza) (2022) – Mitochondrial/ER stress reduction; modest survival benefit.
• Tofersen (2023) – Antisense oligonucleotide for SOD1‑ALS (2% of cases); intrathecal.

Multidisciplinary Supportive Care

• Respiratory – NIV (BiPAP) prolongs survival/quality; tracheostomy for long‑term.
• Nutrition – PEG tube when oral intake insufficient.
• Communication – AAC devices, eye‑gaze tech.
• Mobility – PT, orthotics, power wheelchair.
• Spasticity/cramps – Baclofen, tizanidine, botulinum toxin.
• Sialorrhea – Anticholinergics, botulinum toxin, radiation.
• Palliative care – Early integration improves QoL.

Emerging

• Gene therapies (AAV‑mediated), stem cells, neurotrophic factors, antisense for other genes (C9orf72, FUS, TARDBP).
• Biomarkers (NfL, pNfH) for trial enrichment.

No cure yet; comprehensive clinic care extends survival and dignity.