Motor neuron disorder

This study tested two doses of oral edaravone in 202 people with ALS to see if it could slow disease progression. Participants were followed for up to 48 weeks. The trial was terminated early, so the full results are not available.

This study tested an oral form of edaravone in 384 adults with ALS to see if it could slow the disease's progression over 48 weeks. Participants were randomly assigned to different dosing schedules. The trial was terminated early, and results are not yet fully available.

This early-stage trial tests the safety of siplizumab, a drug that may calm the immune system, in 48 adults newly diagnosed with ALS. Researchers will monitor side effects and how the drug moves through the body. The goal is to see if it is safe enough for larger studies.

This phase 3 trial tested lithium carbonate against placebo in 88 people with ALS to see if it could improve survival and daily function. The study was terminated early, so the full results are not available. The trial aimed to find a treatment that slows the disease, but its ear…

This phase 1 trial tested a gene therapy called AVXS-101 (Zolgensma) in 32 children with spinal muscular atrophy (SMA) who could sit but not stand or walk. The therapy delivers a working SMN gene via a spinal injection to help improve muscle function. The study focused on safety …

This study was designed to let people who had already taken latozinemab in an earlier study continue receiving the drug. Only 17 people took part, and the study was stopped early. The goal was to track safety and how long people stayed on treatment, not to test if the drug could …

This study tested a single dose of STR04, made from a patient's own bone marrow stem cells, for amyotrophic lateral sclerosis (ALS). Only one person enrolled before the study was stopped early. The goal was to see if the treatment was safe and could slow the disease, but no concl…

This study looked at the long-term safety and effectiveness of a daily oral medication called SPG302 in people with ALS who had already taken part in an earlier study. The trial was stopped early, and only 16 people were enrolled. Researchers tracked side effects, suicidal though…

This study tested a new drug called VRG50635 in 54 people with ALS (Lou Gehrig's disease). The main goal was to check if the drug is safe and how the body processes it. The trial was stopped early, but researchers were looking for side effects and changes in disease progression.

This study tested a blood product called immune globulin (Flebogamma) in 191 people with post-polio syndrome, a condition causing new muscle weakness years after polio. The goal was to see if it could improve walking distance and reduce pain. The trial was terminated early, so we…

This study tested an experimental drug called BIIB105 in adults with ALS, a progressive nerve disease. The trial had two parts: a 6-month placebo-controlled phase and a 3-year open-label extension where everyone received the drug. The goal was to check safety, how the body proces…

This early-stage trial tested an experimental drug called DNL747 in 15 people with ALS to see if it was safe and how the body processed it. The study was terminated before completion, so we don't have full results. It was a small, short-term safety study, not a test of whether th…

This pilot study tests whether a short-term group therapy called Emotional Awareness and Expression Therapy (EAET) can help people with persistent physical symptoms like pain or fatigue that have no clear medical cause. The therapy focuses on helping participants identify and exp…

This study tested whether a digital platform (m-Rehab) could help people with ALS who use non-invasive ventilation. The platform included educational content, remote monitoring, and video consultations. The goal was to see if it could slow the loss of physical function and improv…

This study tested whether two gut hormones, GIP and GLP-2, could reduce bone breakdown in children with spinal muscular atrophy, cerebral palsy, or Duchenne muscular dystrophy who use wheelchairs. Participants received a liquid meal and then either a hormone injection or a placeb…

This study looked at how satisfied people with ALS (Lou Gehrig's disease) were when they had doctor visits by video from home, with a nurse or physiotherapist there to help. About 38 patients who used a breathing machine at home took part. The goal was to see if this kind of care…

This study tested an experimental drug called BLZ945 in 28 people with ALS to see if it could reduce brain inflammation. Researchers used special PET scans to measure inflammation. The study was terminated early, so the full results are not available, but it aimed to gather safet…

This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult participants with ALS, FTD, or mixed ALS/FTD phenotype with a documented mutation in the C9orf72 gene. To participate in the…