What the trial was testing
The trial enrolled 176 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 an early-stage trial — researchers are still confirming safety and getting an early look at how well the treatment works. 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
The highest dose of tofersen reduced SOD1 protein levels in spinal fluid by 33%.
The New England journal of medicine · 2020 · NCT02623699
These findings — that the highest dose lowered harmful SOD1 protein in spinal fluid — were published in the The New England journal of medicine and represent the headline result of the study.
Researchers tracked outcomes across 176 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
This was a safety testing study (Phase 1-2) and tofersen was not yet FDA-approved at the time. The drug has since been approved by the FDA in 2023 for adults with ALS caused by SOD1 mutations. If you have SOD1-related ALS, ask your doctor whether tofersen might be right for you.
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
Inspiratory Muscle Training in Amiotrophyc Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS) progressively damages the nerve cells responsible for voluntary muscle movement. Over time, this leads to weakness in different muscles such as those used for movement or breathing. Breathing problems are one of the main causes of complications and reduced survival in people with ALS. This happens because the inspiratory muscles-those that help draw air into the lungs-gradually lose strength. The study has the aim to explore the benefits of training inspiratory muscles in ALS patients in order to maintain the setrength of these muscles for as long as possible and look the impact on respiratory function.
Connect-One: Early Feasibility Study of Connexus® Brain-Computer Interface (BCI)
The Connect-One Study is an early feasibility study to obtain preliminary device safety information for the Connexus Brain-Computer Interface (BCI). The Connexus BCI is intended to be used as: (1) an assistive communication device to decode imagined language correlates and speech for patients with impaired communication as a result of severe loss of voluntary motor control; and (2) to provide control of computer devices for individuals with severe loss of voluntary motor control of the upper extremity.