How Effective Are ALS Medications Riluzole & Edaravone?

Currently, there is no cure for ALS, and FDA-approved medications do not stop the disease from progressing. Instead, these medications are considered disease-modifying therapies, which means their goal is to slow down the speed at which the disease advances ^[1][2]. While they cannot reverse damage or muscle weakness that has already happened, they can offer modest benefits, such as extending life by a few months or helping you maintain physical independence a bit longer ^[1][3].

When considering these medications, it is important to weigh their benefits against the potential side effects, financial costs, and the daily routine required to take them.

Riluzole (Rilutek, Tiglutik, Exservan)

Riluzole was the first medication approved for ALS and remains the standard of care ^[1].

• How effective is it? In clinical trials, riluzole has been shown to modestly extend survival, typically by about 2 to 3 months ^[1][2]. However, some real-world studies suggest this survival benefit might be longer in everyday practice ^[4][5]. Riluzole also helps delay the progression of muscle weakness ^[6].
• How is it taken? It was originally available only as a pill and typically needs to be taken on an empty stomach for proper absorption ^[2]. Because many people with ALS eventually develop dysphagia (difficulty swallowing), riluzole is now also available as a liquid suspension (Tiglutik) and a dissolving film that melts on the tongue (Exservan) ^[7][8].
• What are the side effects? The medication is generally well-tolerated, but it can cause nausea, weakness (asthenia), and shortness of breath ^[9]. Crucially, it can affect your liver, so your doctor will need to monitor your liver function through blood tests closely, especially during the first six months ^[10].

Edaravone (Radicava, Radicava ORS)

Edaravone is designed to relieve oxidative stress—a process where harmful molecules damage cells—in the brain and spinal cord ^[11].

• How effective is it? Edaravone does not clearly extend survival based on current evidence ^[12][13]. Instead, it has been shown to slow the decline of physical function by about 33% over six months ^[3]. This benefit is most clearly seen in patients who are in the early stages of the disease and still have strong breathing function ^[3].
• How is it taken? Edaravone requires a complex “on/off” dosing cycle ^[14]. For the first month, it is taken daily for 14 days followed by 14 days off ^[3]. After that, it is taken for 10 days out of a 14-day period, followed by 14 days off ^[3]. It is available as an intravenous (IV) infusion, which takes about 60 minutes, or as an oral liquid suspension (Radicava ORS) that provides the same medication without needles ^[14][3].
• What are the side effects? Common side effects include bruising, headache, and fatigue ^[15]. Importantly, edaravone carries a risk of severe allergic reactions, including anaphylaxis, and contains sulfites which can trigger dangerous reactions in patients with asthma ^[14]. The main burden for many patients is the strict administration schedule and time commitment for IV infusions ^[14][3].

Targeted Therapies

The landscape of ALS treatment is changing rapidly, but it is important to stay informed about what works and what does not:

• Tofersen (Qalsody): This is a newer, highly targeted therapy available only for a very small percentage of ALS patients who carry a specific genetic mutation called SOD1 ^[16]. Ask your neurologist if genetic testing is appropriate for you. In this specific group, tofersen has been shown to slow disease progression and lower markers of nerve damage ^[16][17]. However, it requires recurrent spinal injections (lumbar punctures) into the fluid surrounding the spinal cord, and about 7% of patients in trials experienced severe neurological side effects like myelitis (spinal cord inflammation) ^[18].
• Sodium Phenylbutyrate/Taurursodiol (Relyvrio): Although approved by the FDA in 2022, this medication was voluntarily withdrawn from the market in 2024 after a large Phase 3 clinical trial (the PHOENIX trial) proved that it did not slow the disease ^[19][20].

Making the Decision

Deciding whether to start an ALS medication is a deeply personal choice. You and your multidisciplinary ALS care team should have an open conversation about your goals. Because ALS medications can be incredibly expensive and difficult to get approved by insurance, be sure to ask your care team about financial assistance programs ^[2].

Some patients choose to take every available medication to buy as much time and function as possible. Others, particularly in the later stages of the disease, may decide that the financial burden, frequent dosing schedules, blood tests, and side effects outweigh the modest benefits. They may choose instead to focus entirely on managing symptoms and maximizing day-to-day comfort. Both are completely valid paths.