Marco Meglio

Enrollment for the phase 1/2 study will be partially stopped; however, all participants included in the trial, whether on AOC 1001 or placebo, are eligible to continue their current dosing regimen.

Using magnetic resonance spectroscopy, investigators observed significantly different levels of total NAA across MS and NMOSD subtypes, and a negative correlation between total NAA and disability score.

When hydromethylthionine mesylate was compared with publicly available placebo decline data, the treatment effects of cognitive and functional decline are about 3-fold larger over 18 months.

Nicholas Silvestri, MD, clinical professor at the University at Buffalo discussed topics related to the expansion of treatments for myasthenia gravis and the positive outlook of managing the disease.

The clinical program manager at the Jefferson Center for Neurorestoration discussed a new approach to restoring arm function using a powered arm brace and muscle stimulation system.

No statistical differences were found between REN and the standard of care medications, in any of the effectiveness outcomes of pain single-treatment pain relief and pain freedom, nor in consistency of pain relief and pain freedom.

Despite not meeting its primary and secondary end points, CNM-Au8 will continue to be evaluated in an open-label extension in HEALEY ALS and could potentially be offered in an expanded access protocol program.

Designed to treat the underlying cause of FSHD, AOC 1020 will be evaluated on safety, tolerability, pharmacokinetics, and pharmacodynamics in a cohort of 68 adults with the disease.

Justin Klee and Josh Cohen, cofounders and cochief operating officers, Amylyx Pharmaceuticals, answered questions related to the new approval of AMX0035 for ALS and how it changes the treatment landscape going forward.

Sleep changes experienced by patients on apomorphine infusion were indicated by scores on Insomnia Severity Index and Clinical Global Impression-Improvement Scale.

Despite not improving clinical end points, treatment with tofersen was associated with reduced in the total concentration of SOD1 protein and neurofilament light chain, a marker of axonal injury.

In additional data from CENTAUR, Amylyx touted the drug's ability to prolong tracheostomy-free and ventilation-free survival.

After previously failing a phase 3 trial of patients with multiple system atrophy, verdiperstat continued to show an effect in patients with ALS that was similar to those on placebo.

Ashgan Elshinawy, DO, a pulmonologist at Penn Medicine, assessed the current ways to treat patients with insomnia and the areas of needed improvement.

Consistent with the overall population, newly diagnosed, treatment-naïve patients with MS showed better relapse rates, all-cause disability worsening—including progression independent of relapse activity—with ofatumumab over teriflunomide.

The professor of neurology and pediatrics at the University of Rochester Medical Center provided insight on the emerging therapies and advancements for treating patients with Duchenne muscular dystrophy.

Avindra Nath, MD, clinical director of the National Institute of Neurological Disorders and Stroke, provided insight on new findings that may explain the symptoms of certain cases of sporadic ALS.

Over a 3.5-year treatment period, the risk of serious infections or malignancies, few of which were previously reported, did not increase with additional ofatumumab exposure.

Over a long-term follow-up, a high proportion of patients were compliant to therapy, with adverse events mild-to-moderate in nature, regardless of individuals being on continuous treatment or switched from placebo.

Brian Callaghan, MD, MS, associate professor at the University of Michigan, discussed his presentation at AANEM 2022 on changing the treatment paradigm for painful diabetic neuropathies.

Among a surveyed cohort of patients with myasthenia gravis, 75% reported that their work was impacted, and 27% of respondents claimed they lost wages as a result of MG.

Minimal symptom expression, demonstrated by scores of 0 to 1 on MG-ADL, was observed in more than 25% of both rozanolixizumab groups compared with 3.0% of those on placebo.

More than 85% of the individuals who continued givosiran (Givlaari; Alnylam) through the 36-week open-label extension were attack free in the final 3 months of treatment.

From the pre-COVID period to 6 weeks after vaccination, the reporting rate of GBS was significantly different, regardless of whether Brighton criteria was applied to the analysis. The authors noted that passive surveillance limitations warrant further analysis.

After 12 weeks of treatment with zilucoplan 0.3 mg/kg, almost three-fourths of patients demonstrated at least a 3-point reduction in Myasthenia Gravis Activities of Daily Living scores.

The director of NYU Langone’s Alzheimer’s Disease Research Center and Center for Cognitive Neurology provided perspective on raising awareness for World Alzheimer’s Day and the ongoing challenges patients with the disease face.

After demonstrating a positive reduction of mutant huntingtin protein in preclinical studies, WVE-003 continued to show similar results when assessed in 30- and 60-mg doses.

One of the identified loci related to clinical progression in Parkinson disease expresses ADORA2A in the cerebellum, which encodes the adenosine A2A receptor, a promising target for therapeutics in PD.

The Parkinson disease agent showed a safe and tolerable profile, with significantly greater improvements in MDS-UPDRS-III scores and OFF time per day.

The explosion of new science and legislative decisions have expanded the capabilities and reach of newborn screening, providing thousands of families every year an opportunity to seek early, and critical, care for their infants.