The NeurologyLive® neuromuscular disease clinical focus page offers updates and information related to the clinical care and management of individuals with neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, cerebral palsy, and spinal muscular atrophy, as well as rare diseases. This page consists of news, podcasts, and videos about the latest FDA actions, clinical guideline updates, interviews with physicians, study and clinical trial findings, and more.
August 29th 2025
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 29, 2025.
Rituximab Significantly Lessens Myasthenia Gravis Manifestations, Rescue Treatments Needed
November 18th 2022Compared with placebo, a greater proportion of patients on rituximab met the primary end point of minimal disease manifestations at 16 weeks, and demonstrated favorable results on several secondary end points.
NeuroVoices: Timothy Miller, MD, PhD, on the Impact of Tofersen, Antisense Therapeutics in ALS
November 16th 2022The codirector of the ALS Center at Washington University School of Medicine in St. Louis provided perspective on the phase 3 VALOR findings of tofersen, an antisense agent currently under review to treat SOD1-mutated ALS.
Critical Takeaways, Learning Experiences From the Tofersen Trials in ALS: Timothy Miller, MD, PhD
November 11th 2022The codirector of the ALS Center at Washington University School of Medicine provided perspective on the lessons learned from the trials of tofersen, as it aims to become the first FDA-approved therapy specific to SOD1 ALS. [WATCH TIME: 4 minutes]
Role of Tofersen Among Other Previously Approved ALS Therapies: Timothy Miller, MD, PhD
November 10th 2022The codirector of the ALS Center at Washington University School of Medicine in St. Louis provided background on how tofersen would be used alongside other ALS medications if approved. [WATCH TIME: 3 minutes]
Use of Nanoparticles to Combat Neurodegenerative Disease
November 10th 2022Available neurodegenerative disease treatments are generally unsafe and ineffective at penetrating the blood-brain barrier, though the use of nanoparticles can provide improved penetration and exert a neuroprotective effect.
NurOwn Stromal Cell Therapy Continues to Show Positive Data in Less Severe ALS
November 9th 2022After removing participants at higher risk of reaching a floor effect of the ALSFRS-R, those treated with NurOwn demonstrated a higher rate of clinical response and less function lost across 28 weeks than placebo.
Advocacy and Empowerment: Finding Your Advocacy Community
November 9th 2022The Palatucci Advocacy Leadership Forum, or PALF, sponsored by the American Academy of Neurology, gives neurologists and trainees tools to successfully advocate for their ideas and develop their identity as physician advocates.
Understanding Tofersen’s Impact on SOD1 ALS, Neurofilament Light: Timothy Miller, MD, PhD
November 9th 2022The codirector of the ALS Center at Washington University School of Medicine in St. Louis discussed the long-term data of VALOR and its open-label extension assessing tofersen (Biogen) in SOD1 ALS. [WATCH TIME: 4 minutes]
Patient-Reported Outcomes Worsen as ALS Disease Progresses, Real-World Study Shows
November 4th 2022On King’s staging systems, ALSAQ-5 scores increased from 24.6 at stage 1 to 62.1 at stage 4, whereas for Milano-Torino Staging systems, patients’ ALSAQ-5 scores increased from 43.2 at stage 0 to 80.7 at stage 3.