What the trial was testing
The VALOR enrolled 139 patients with als. The study was sponsored by Biogen and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Tofersen lowered harmful SOD1 protein levels in spinal fluid but didn't improve physical function in the first 28 weeks.
The New England journal of medicine · 2022 · NCT03070119
These findings — that people who started tofersen right away had slightly better muscle function after one year than those who waited six months — were published in the The New England journal of medicine and represent the headline result of the study.
Researchers tracked outcomes across 139 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with als, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Tofersen was FDA-approved in 2023 for adults with ALS linked to SOD1 mutations, though it works differently than traditional treatments. The drug requires spinal injections and can cause side effects including headaches and falls. If you have SOD1 ALS, talk with your doctor about whether tofersen is right for you and what to expect from treatment.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open als trials
Evaluation of the Combined Therapy of EH-301 and N-acetylcysteine Together With Riluzole in Amyotrophic Lateral Sclerosis (ALS)
This study is designed to evaluate whether a combination of N-acetylcysteine (NAC) and EH-301 can slow down or improve symptoms of amyotrophic lateral sclerosis (ALS). Researchers will assess changes in disease progression using the ALS Functional Rating Scale-Revised (ALSFRS-R), a standard tool for measuring daily functioning in people with ALS. The main question is whether taking NAC together with EH-301 can prevent symptom worsening and possibly improve existing ALS symptoms. Participants will be randomly assigned to receive either the active combination (NAC + EH-301) or matching placebos for 6 months. During this period, they will attend regular clinic visits for evaluations, tests, and safety monitoring. After completing the initial 6-month phase, all participants may choose to join a 6-month open-label extension, where everyone receives the active treatment regardless of their original group.
UPenn Observational Research Repository on Neurodegenerative Disease
The aim of this study is to create a repository of both cross-sectional and longitudinal data, including cognitive, linguistic, imaging and biofluid biological specimens, for neurodegenerative disease research and treatment.