← Back to news
ResearchRSSWednesday, April 22, 2026 · 3 days ago

Antipsychotic drug may help treat SMA symptoms, research shows

WHY IT MATTERS

If haloperidol proves effective in human trials, SMA patients could potentially benefit from a medication that already exists and is FDA-approved, potentially offering a faster path to treatment than developing entirely new drugs.

Scientists found that haloperidol, a medication normally used to treat psychiatric conditions, may help treat spinal muscular atrophy (SMA) by increasing levels of a protein called SMN that is missing or low in SMA patients. In laboratory tests with mouse cells and human patient cells, haloperidol helped nerve cells survive longer, reduced harmful inflammation, and improved movement. This suggests haloperidol could potentially be used alongside or instead of current SMA treatments.

The antipsychotic medication haloperidol may help treat spinal muscular atrophy (SMA) by increasing SMN protein levels and enhancing protective effects in nerve cells, a study found. Haloperidol boosted nerve cell survival, reduced neuroinflammation, and improved motor function in mice and in patient-derived cells, supporting its potential as a complementary or stand-alone therapy. “Given its central […] The post Antipsychotic drug may help treat SMA symptoms, research shows appeared first

ASK YOUR DOCTOR

Ask your SMA specialist or neurologist at your next appointment whether haloperidol might be appropriate to discuss as a potential complementary treatment, and whether any clinical trials testing this approach are available in your area.

Find a specialist →Learn more ↗
smaneuroprotectiondrug repurposingpreclinical researchsmn protein

Related conditions

Spinal muscular atrophy associated with central nervous system anomalySpinal muscular atrophy with respiratory distress type 2Spinal muscular atrophy with respiratory distress type 1