Neuromuscular disease

This early-stage study tested a new drug called PF-07284892, alone or with other medicines, in people with advanced solid tumors that had specific genetic changes. The goal was to find the safest dose and check for side effects. The study was stopped early, so results are limited…

This phase 2 study tested an experimental oral drug called BGB-45035 in 49 adults with moderate to severe rheumatoid arthritis who had not responded well to standard treatments. Participants received either the drug or a placebo to see if it could reduce joint pain and swelling. …

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 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 different warming methods—like forced air blankets and warmed IV fluids—to prevent hypothermia in women having planned C-sections. Only 16 women participated before the trial was stopped early. The goal was to see which method best keeps mother and baby warm and…

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 study tested the safety of golodirsen (Vyondys 53) in boys and men with Duchenne muscular dystrophy who can no longer walk. Only 2 people took part before the trial was stopped early. Participants received weekly IV infusions for up to 96 weeks, with extra follow-up. The goa…

This study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …

This study tested whether high-dose fish oil supplements could improve nerve function in people with prediabetes and early diabetic neuropathy. Researchers planned to enroll 40 adults aged 55–80, but the trial was terminated early with only 31 participants. Participants took eith…

This Phase 3 trial tested an oral drug called venglustat in 75 adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that cause progressive nerve damage. The drug aimed to lower toxic fat buildup in the brain and slow disease worsening. However…

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 gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E (LGMD2E), a rare genetic disease that causes muscle weakness. The treatment aimed to deliver a working gene to muscle cells to help them produce a missing protein. Only 6 peopl…

This early-phase trial tested whether nicotinamide riboside (a form of vitamin B3 sold as Niagen) could help repair nerve damage in people with type 2 diabetes or prediabetes. Only 10 participants were enrolled before the study was terminated. The main goal was to measure changes…

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 losmapimod in 14 adults with FSHD1, a rare genetic condition that causes progressive muscle weakness. The main goal was to check safety and tolerability, and to see if the drug affects certain biological markers. The trial was termina…

This phase 2 study tested a drug called PGN-EDO51 in 7 people with Duchenne muscular dystrophy whose genetic mutation can be corrected by skipping exon 51. The drug was given by IV infusion to see if it is safe and tolerable. The trial was terminated, so results are limited.

This study tested a gene therapy (delandistrogene moxeparvovec) combined with a drug called imlifidase to see if it could safely deliver the therapy to boys with Duchenne muscular dystrophy who had antibodies that might block the treatment. Only 5 participants were planned, but t…

This study tested a drug called losmapimod for people with a rare muscle-weakening disease called FSHD. The goal was to see if the drug could slow muscle loss and improve arm function over 48 weeks. About 260 adults with FSHD were randomly assigned to receive either losmapimod or…

This early-stage trial tested a gene therapy called SBT101 for adrenomyeloneuropathy (AMN), a rare nerve disease that causes walking difficulties. Eight adults received either the therapy or a sham procedure. The study was terminated early, so we have limited data on safety and e…

This early-phase trial tested a new immunotherapy approach for children with DIPG, a rare and aggressive brain stem tumor. After standard radiation and chemotherapy, patients received special vaccines and immune cells designed to attack the tumor. The study was small (11 particip…

This Phase 2 trial tested a single dose of gene therapy (fordadistrogene movaparvovec) in 10 boys with early-stage Duchenne muscular dystrophy. The goal was to check safety and whether the therapy could help muscles produce a mini-dystrophin protein. The study was terminated earl…

This study follows 7 people with Duchenne muscular dystrophy who previously received an experimental gene therapy called fordadistrogene movaparvovec. Researchers will monitor them for 10 years to check for side effects and see if the treatment continues to help with movement. Th…

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 phase 2 trial tested the drug losmapimod in 76 adults with FSHD, a genetic condition that causes progressive muscle weakness. Participants took either losmapimod or a placebo for 48 weeks to see if the drug was safe and could help control the disease. The study was terminate…

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 phase 3 trial tested the drug miglustat (Zavesca) in 30 infants with Sandhoff or Tay-Sachs diseases, rare genetic disorders that destroy nerve cells. The goal was to see if the drug could reduce hospitalizations, seizures, and feeding problems while improving motor function.…

This early-stage trial tested a single infusion of gene therapy (PF-06939926) in 23 people with Duchenne muscular dystrophy, both those who could still walk and those who could not. The main goal was to check safety and tolerability, while also measuring dystrophin protein levels…

This early study tested whether a blood-cleaning procedure called plasmapheresis could allow boys with Duchenne muscular dystrophy who have antibodies against the gene therapy carrier to still receive the treatment. Only 3 boys were enrolled before the study was stopped early. Th…

This was a very early (Phase 1) study testing a gene therapy called SRP-6004 for people with limb girdle muscular dystrophy type 2B/R2, a rare muscle-weakening disease. The goal was to see if a single IV infusion of the therapy is safe and can help the body produce a missing prot…

This early-stage trial tested a gene therapy called SRP-9004 in just 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, not effectiveness. The study was terminated early, so results are limited.

This early-stage trial tested a gene therapy called AXO-AAV-GM2 in children with Tay-Sachs or Sandhoff disease, rare and fatal genetic brain disorders. The therapy delivers healthy genes directly into the brain and spinal fluid to try to restore a missing enzyme. The study was te…

This study compared a new, minimally invasive ultrasound-guided procedure to standard surgery for carpal tunnel syndrome. The goal was to see if the new technique could provide similar relief with a smaller incision and faster recovery. Only 7 people were enrolled before the stud…

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 trial tested whether a TENS device, which sends mild electrical pulses to the skin, can help relieve nerve pain caused by chemotherapy in early-stage breast cancer patients. The study enrolled 17 people who were having taxane chemotherapy and had at least mild nerve pain in …

This study looked at whether adding platelet-rich plasma (PRP) to standard carpal tunnel release surgery helps people with severe carpal tunnel syndrome. The trial planned to enroll 15 adults with severe nerve damage. It compared surgery with PRP to surgery alone. The study was t…

This study tested a soft-robotic glove called Carbonhand in 6 people with hand weakness due to aging or neuromuscular disease. The glove supports finger movement to help with daily tasks. Researchers measured how the glove affected movement, pain, and fatigue during strenuous act…

This study tested a device called Exablate, which uses focused sound waves to treat areas of the brain linked to pain. The goal was to see if it could reduce pain and improve quality of life in people with chronic nerve pain. Only 2 people enrolled before the study was stopped, s…

This study tested whether a 12-week program of walking and resistance exercises or nutrition education could reduce nerve pain caused by platinum chemotherapy. The trial enrolled 36 adults with cancer who were receiving oxaliplatin or cisplatin. The study was terminated early, so…

This study tested an experimental drug called RTA 901 to see if it could safely reduce nerve pain in people with diabetes. The trial planned to enroll 209 adults with diabetic peripheral neuropathic pain, comparing two doses of the drug against a placebo over 12 weeks. However, t…

This study aimed to find the best amount of exercise needed to improve dizziness symptoms in people with balance disorders. Researchers used virtual reality (VR) to measure how much exercise helps and compared VR exercises to standard physical therapy. The study included people w…

This study tested an experimental drug called GSK3858279 in 147 adults with chronic nerve pain from diabetes. The goal was to see if it could reduce pain better than a placebo. However, the trial was terminated early, so we have less information than planned.

This study tested a device called BurstDR spinal cord stimulation to help people with painful diabetic neuropathy that didn't get better with standard medicines. The goal was to see if it could cut leg pain by at least half. Only 6 people joined before the study was stopped early…

This study tested a device called SinuSonic that uses sound vibrations and pressure to relieve sinus headache pain. The trial included 27 adults with facial pain lasting more than 3 months. Participants used either the real device or a sham device that looked the same but didn't …

This early-stage study aimed to compare how a tablet form of tafamidis is absorbed in the body compared to the existing capsule form. It involved 24 healthy adults who took a single dose of each form under fed conditions. The study was terminated early, so results may be limited.

This study looked back at 20 young people who came to the hospital with nerve problems after using nitrous oxide (laughing gas) for fun. Researchers measured muscle strength, balance, and disability to understand how the gas harms the nervous system. The goal was to learn more ab…

This study tested whether special insoles with pressure sensors (FEETME®) can accurately measure walking speed in people with CIDP, a nerve condition that causes walking difficulties. Researchers compared the insoles to a standard walkway system in 18 adults aged 18-75 with CIDP.…

This study tested a new method called cortical vestibular evoked potentials (C-VEPs) to measure how the brain processes balance signals from the inner ear. Researchers compared people with normal balance to those with vestibular neuronitis (a cause of vertigo) to see if the test …

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 looked at whether people born with cytomegalovirus (CMV) infection who have hearing loss or balance problems also have a condition called endolymphatic hydrops (fluid buildup in the inner ear). Researchers used a special MRI scan with a contrast dye to check for this f…

This study followed 65 adult men with a rare inherited nerve disease called AMN (a form of spastic paraplegia) to understand how their symptoms change over time. Researchers collected data on walking ability and quality of life. The goal was to fill gaps in knowledge about the di…

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 study looked at muscle samples from 7 children with brachial plexus birth palsy who had shoulder stiffness. Researchers wanted to understand why the shoulder gets stiff again after surgery, by checking for muscle wasting and scarring. The study was terminated early, so findi…

This study tested a new 3D scanning device that aims to help surgeons match a patient's spine bones to their MRI or CT scans during surgery. The goal was to see if the scanner could accurately and consistently map the spine to improve surgical precision. Only one person took part…

This study aims to find out how common transthyretin amyloidosis cardiomyopathy (ATTR-CM) is in Russian patients with a certain type of heart failure. Researchers will review medical records and then invite some patients for extra heart tests to confirm or rule out ATTR-CM. The g…

This study looked at one person with facioscapulohumeral muscular dystrophy (FSHD) to better understand the disease. Researchers examined muscle tissue and checked for specific biomarkers. The goal was to learn more about how FSHD affects the body, not to test a treatment.

This study follows up on children with Krabbe disease who received a one-time gene therapy infusion (FBX-101) in earlier trials. Researchers will monitor safety and measure motor skills over time. Only 2 participants are enrolled, so results are very limited.