We need more – likely many more – effective treatments to end ALS. Progress in ALS research has been too slow for too long but, in 2024, we’re witnessing significant advances and a new pace of progress. The past few years have see more treatments for ALS in clinical trials than ever before. Still, most drugs that reach trial fail, in any disease. However, the more experimental treatments that make it to trials – with good preclinical evidence to support them – the more likely there are to be successes.

Recently, we have even seen approvals for new ALS treatments, such as tofersen, a genetic treatment developed by Biogen for SOD1-related ALS, sold under the brand name Qalsody. Dr. Stephanie Fradette was one of the people behind milestone. As the VP Head of Neuromuscular Development at Biogen, she oversees the company’s research and development of treatments for ALS and related diseases.

Today, on Endpoints, Dr. Fradette joins us to talk about the current state of ALS research ­- the biggest challenges facing researchers, the developments that give her hope for the future, and how the ALS research community could better serve people with the disease.

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