Neuromuscular

The Child Neurology Foundation is using community outreach and a new social services network to help children with neurological conditions feel included in contemporary society.

The 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]

NurOwn, otherwise known as autologous mesenchymal stromal cells secreting neurotrophic factors cells, has demonstrated significant effects on disease progression in less severe forms of ALS.

Available 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.

After 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.

The 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.

The 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]

The trial, launched through a partnership between Cure Rare Disease and UMass Chan Medical School, assessed CRD-TMH-001, a CRISPR-based gene therapy.

Here's some of what is coming soon to NeurologyLive® this week.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is headache and migraine.

Those who reported higher ALSFRS-R speech scores at baseline had significantly higher diadochokinetic and word rate than those who reported speech disturbances, or lower scores.

By comparing results with pooled placebo groups from the PRO-ACT database, the data showed a 67% slower ALSFRS-R progression with IPL344.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 4, 2022.

On 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.

Nearly one-fourth of the observed treatment-emergent adverse events were related to edaravone. Additionally, there were no serious TEAEs found, and TEAEs were mostly representative of ALS progression.

A retrospective study presented at the 2022 Annual NEALS Meeting reported health disparities in the initial presentation of Black patients with ALS in comparison with White patients.

Treatment with RNS60 resulted in significantly slower rate of decline in FVC and eating and drinking domains of the ALSAQ-40 scale over a 24-week period.

Treatment with tegoprubart resulted in significant reductions of CD40L and CXCL13 levels that occurred after first infusion and sustained throughout the treatment period.

As patients with ALS may have altered taste perception and have expressed that bitterness is associated with AMX0035 (Relyvrio; Amylyx) administration, a number of coping strategies have suggested a workaround to ensure treatment adherence and effectiveness.

Christina Fournier, MD, MSc, associate professor of neurology, Emory University, discussed her presentation at the 2022 NEALS Consortium using a telemedicine exam to track ALS progression.

Once thought nearly impossible to treat, ALS has seen another step of progress with the approval of AMX0035 and a future of potential therapeutics on the way.

SBT-272, an investigational small molecule in development for ALS and other neurological disease of mitochondrial dysfunction, resulted in improved membrane potential and axonal outgrowth of TDP-43 in vitro.

The adult neurologist at Allegheny Health Network provided perspective on why there’s never been a better time to treat patients with myasthenia gravis.

Here's some of what is coming soon to NeurologyLive® this week.

The PhD student at Aarhus University provided insight on his study presented at 2022 ECTRIMS Congress on an innovative way using neuromuscular measures to discriminate those with MS at high risk for falls. [WATCH TIME: 2 minutes]