Marco Meglio, Editor for NeurologyLive, has been with the team since October 2019. Follow him on Twitter @marcomeglio1 or email him at email@example.com
Nerivio Remote Electrical Neuromodulation Could Serve as Alternate Treatment for Chronic Migraine
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.
CNM-Au8 Shows Survival Benefit in HEALEY ALS Trial, But Fails to Meet End Points
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.
Trial Launched for Avidity’s AOC 1020 in Facioscapulohumeral Muscular Dystrophy
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.
Reactions, Ramifications, and Forward Progress of Newly Approved AMX0035 for ALS
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.
Night-Time Only Apomorphine Infusion Shows Benefit to Treating Parkinson Disease Insomnia
Sleep changes experienced by patients on apomorphine infusion were indicated by scores on Insomnia Severity Index and Clinical Global Impression-Improvement Scale.
Open-Label Extension Data Highlight Tofersen’s Ability to Reduce SOD1 Protein in ALS
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.
AMX0035 Approved for the Treatment of ALS
In additional data from CENTAUR, Amylyx touted the drug's ability to prolong tracheostomy-free and ventilation-free survival.
Verdiperstat Fails to Differentiate From Placebo in Critical HEALEY ALS Platform Trial
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.
State of Insomnia Care and Overcoming Inconsistencies in the Traditional Care Strategies
Ashgan Elshinawy, DO, a pulmonologist at Penn Medicine, assessed the current ways to treat patients with insomnia and the areas of needed improvement.
Ofatumumab Outperforms Teriflunomide in Newly Diagnosed, Treatment-Naïve Multiple Sclerosis
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.
NeuroVoices: Emma Ciafaloni, MD, on the Vast Expansion of Innovative Approaches to Duchenne Muscular Dystrophy
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.
Unraveling the Roots of ALS Through Reactivation of HERVs and Antibody Levels
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.
Long-Term Safety of MS Treatment Ofatumumab Confirmed in ALITHIOS Extension
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.
Dimethyl Fumarate Shows Low MS Relapse Rates, Encouraging Safety After 13-Year Analysis
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.
Understanding New Guidelines to Improve Management of Painful Diabetic Neuropathies
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.
Substantial Economic Burden Is Frequent in Myasthenia Gravis, Primarily Driven by Direct Costs
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.
Rozanolixizumab Continues to Show Efficacy, High Responder Rates in Myasthenia Gravis
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.
Givosiran Shows Long-Term Clinical Benefit in Acute Hepatic Porphyria, Open-Label Findings Suggest
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.
Potential Temporal Association Found Between Guillain-Barré Syndrome and COVID-19 Vaccination in Pediatrics
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.
High Proportion of Zilucoplan Responders Identified in Secondary Analysis of RAISE Trial
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.
NeuroVoices: Thomas M. Wisniewski, MD, on Raising Awareness for World Alzheimer’s Day
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.
WVE-003 Demonstrates Huntington-Specific Biomarker Changes in Phase 1b/2a Study
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.
Several Genetic Loci Show Increased Risk of Mortality, Cognitive Impairment in Parkinson Disease
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.
Selective Adenosine A2A Antagonist KW-6356 Demonstrates Efficacy, Safety as Adjunct to Levodopa in Parkinson Disease
The Parkinson disease agent showed a safe and tolerable profile, with significantly greater improvements in MDS-UPDRS-III scores and OFF time per day.
Celebrating the Expansion and Stressing the Importance of Newborn Screening
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.
Multiple-Source Constant-Current DBS Improves Quality of Life in Parkinson Disease, Real-World Analysis Suggests
Over a 2-year period, patients demonstrated improvements in Parkinson’s Disease Questionnaire scores, along with a 35% improvement in MDS-UPDRS-III scores.
NurOwn Treatment Shows Positive Results in Phase 2 Study of Progressive Multiple Sclerosis
Treatment with NurOwn resulted in response on several secondary outcomes, including timed 25-foot walk, 12-item multiple sclerosis walking scale, and low contrast letter acuity, among others.
Trauma and Discrimination Worsen Migrainous Headache in Sexual and Gender Minority Individuals
Approximately half of the respondents with a history of headache screened positive for migrainous headache by ID-migraine criterion with most reporting moderate to severe intensity headache.
COVID-19 Headache Positively Associated With Mortality, Meta-Analysis Suggests
In addition to headache, other COVID-19 symptoms such as anosmia, myalgia, and nausea or vomiting were associated with higher survival rates.
Increased Tau Burden Observed in Abnormal Central Vascular Hemodynamics
Patients who demonstrated increased central pulse pressure and forward wave amplitude had greater entorhinal tau burden. These measures were not associated with amygdala, inferior temporal, nor precuneus tau burden.
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