Neuromuscular

Jennifer Roggenbuck, MS, LGC, an associate professor of neurology and internal medicine at The Ohio State University Wexner Medical Center, discussed her presentation on evidence-based consensus guidelines for genetic testing and counseling from the 2022 NEALS Consortium.

Neurology News Network for the week ending November 12, 2022. [WATCH TIME: 4 minutes]

James Wymer, MD, FAAN, discussed his presentation at the 2022 Annual NEALS meeting on improving speech and swallowing using Neudexta for patients with ALS.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 11, 2022.

The 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]

A phase 1/2a clinical trial resulted in showing that a single dosing of AstroRx was safe and tolerable, at either a low or high dose, for patients with ALS.

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.