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Articles

Recent results from a phase 2 trial of ATH434 revealed its potential in reducing brain iron accumulation and preserving motor function in early-stage multiple system atrophy.

Multiple System Atrophy Agent ATH434 Shows Promising Topline Efficacy in Phase 2 Trial

Findings from previous phase 3 trial showed that treatment with AXS-07 resulted in significant reductions in headache pain freedom, migraine symptoms, and rescue medication use.

FDA Approves Axsome Therapeutics’ AXS-07 for Migraine Treatment

The initial cohort of the EPISOD1 trial demonstrated no significant safety concerns for AMT-162, enabling progression to the next dose-escalation phase.

Phase 1/2 EPISOD1 Trial of SOD1 Agent AMT-162 to Proceed Following Favorable Recommendation

A new study identified key clinical and ultrasound markers that improved the differentiation between 2 conditions with overlapping features.

Clinical and Ultrasound Scores Helps Differentiate CIDP from Diabetic Polyneuropathy

Associate Chair of Pediatrics, and Director, Powell Gene Therapy Center, University of Florida; Chief Medical Advisor, Muscular Dystrophy Association

Videos

The chief medical advisor at the Muscular Dystrophy Association shared key highlights of what attendees can expect at the 2025 MDA Clinical and Scientific Conference.  [WATCH TIME: 4 minutes]

What to Expect at MDA’s 2025 Clinical and Scientific Conference: Barry J. Byrne, MD, PhD

Assistant professor of pediatrics at the University of Michigan.

A duo of experts discussed the importance of addressing barriers such as provider comfort, access to genetic counselors, and insurance coverage to reduce diagnostic latency and standardize genetic testing for epilepsy. [WATCH TIME: 5 minutes]

Insights From the Pediatric Epilepsy Research Consortium on Advancing Genetic Testing

William S. and Lois Stiles Edgerly Professor of Neurology and Professor of Immunobiology; Chair, Neurology; Neurologist-in-Chief, Yale New Haven Hospital

The William S. and Lois Stiles Edgerly Professor of Neurology at Yale School of Medicine talked about refining multiple sclerosis in line with the updated diagnostic criteria. [WATCH TIME: 5 minutes]

Enhancing Multiple Sclerosis Diagnosis and Early Treatment Strategies: David A. Hafler, MD, FANA

Clinical Pharmacy Specialist -CNS, The University of Cincinnati Medical Center; Adjunct Assistant Professor, The James L. Winkle College of Pharmacy, University of Cincinnati and Community of Scholars Faculty, The University of Cincinnati College of Medicine

Kiranpal Singh Sangha, PharmD, clinical pharmacy specialist and adjunct assistant professor of pharmacy at The University of Cincinnati, talked about the vital role of clinical pharmacists in neurology, specifically in multiple sclerosis care.

The Role of Clinical Pharmacists in Neurology: Focus on Neuroimmunology and Multiple Sclerosis Care

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The Psychiatrist at Psychiatric Services Solothurn and University of Basel discussed how heart rate variability correlates with sleep stages, which may indicate nocturnal brain activity. 

Thorsten Mikoteit, MD: Sleep Research, an Excellent Source for Biomarkers

Under treatment, the dynamic of REM sleep related heart rate variability shows early changes during the first week, providing a promising biomarker of treatment.

Thorsten Mikoteit, MD: Sleep Biomarkers in Insomnia

In a recently published retrospective, comparative study, investigators found significant gray matter differences, with the thalamus consistently smaller in patients with multiple sclerosis (MS) as compared to those with neuromyelitis optica spectrum disorder (NMOSD). While not groundbreaking, these findings further emphasized the importance of advanced imaging technologies in differentiating the 2 disorders, which may also have implications for diagnosis and treatment strategies.

The study, which took place at King Saud University Medical City, Saudia Arabia, comprised of 24 patients with NMOSD and 25 patients with MS who were both on immunomodulatory treatments. To be included, subjects must have had at least 2 MRI scans, including T1-weighted FSEGE sequences, with follow-ups performed at least 6 months apart. During the study, investigators examined temporal changes between the 2 cohorts, including analyses of anatomical T1-weighted MRI scans using the CAT12 toolbox.

Led by Salman Aljarallah, MBBS, assistant professor at King Saud University in Saudi Arabia and serves on the medical advisor board at 

, results showed that cerebral white matter (excluding infratentorial white matter) was significantly larger in patients with MS compared with those with NMOSD (69.3 [±10.3] vs 62.8 [±9.3]; 

= .02). More notably, between the 2 groups, there was smaller thalamic size volume in patients with MS compared with NMOSD at baseline (MS average: 6.8 [±3.0]; NMOSD: 9.2 [2.6]; 

When parsing patients with NMOSD by subtype, results showed that those with seronegative NMOSD (n = 5) had the largest thalamic volume (average, 10.96 mL [±2.18]) followed by MOG-positive NMOSD (average 9.1 mL [±2.3]) and aquaporin-4-positive patients (8.6 mL [±2.8]). This difference between average thalamic volume between MS and NMOSD groups persisted in follow-up MRI (6.8 [±3.2] and 8.7 [±2.6], 

Air Pollution Worsens Outcomes in MS and NMOSD, Study Finds

"This study identified that, indeed, significant differences do exist between MS and NMOSD, even if clinical presentations are more or less similar,” Aljarallah et al wrote.

 "While some of these differences, including gross abnormalities such as plaques, can be easily visually seen in routine MRI, we identify more subtle differences in the deep gray matter volume that help differentiate MS from NMOSD, which helps in the management. The most consistent finding in this study was that a smaller thalamic volume in patients with MS was a strong predictor for the diagnosis of multiple sclerosis and is a good marker to differentiate MS from NMOSD."

Receiver under the curve (ROC) analyses revealed differentiated thalamic volume between the 2 groups, with a 70.6% sensitivity, 64.3% specificity, and an area under the operating curve of 0.73, thus identifying a volume less than 7.06 mL as favoring MS. Cerebral white matter volume was larger in MS (OR, 0.9; 

= .03), but this lost significance after adjusting for age (OR, 0.97; 

= 0.4). Furthermore, caudate volume was smaller in MS on follow-up scans, with reduced volume linked to higher odds of MS (OR, 2.5; 95% CI, 1.03-6.25; 

At the 6-month follow-up, on average, both patients with MS and NMOSD demonstrated some reduction in gray matter volume, caudate volume, and cerebellar volume; however, these differences were not statistically significant. Overall, patients with MS had lower whole brain volume, cerebral white matter volume, and hippocampal at follow-up, while patients with NMOSD had lower cerebrospinal fluid volume and thalamic volumes at follow-up compared with baseline. At baseline, all patients with MS had optic neuritis, compared with 75% of those with NMOSD. In addition, patients with MS more often had transverse myelitis (64% vs 41%) and cerebral or brainstem symptoms.

The study had a small number of limitations, including the fact that the sample size was not large in size and the study was single-centered. While the study excluded patients who had MRIs done using different protocols, this may have made the results more representative of a real-world population. Also, the study did not include patients with MOG antibodies who did not fulfill the criteria of NMOSD.

REFERENCE
1. Alosaimi M, Alkanhal H, Aldeligan S, et al. Thalamic volume differentiates multiple sclerosis from neuromyelitis optica spectrum disorder: MRI-based retrospective study. 

Front Neurol Multiple Sclerosis and Neuroimmunology. 

2025;2(15). doi:10.3389/fneur.2024.1491193

A comparative study at King Saud University Medical City identified significant differences in gray matter between multiple sclerosis (MS) and neuromyelitis optica spectrum disorder (NMOSD) patients, particularly in thalamic volume. MS patients exhibited smaller thalamic volumes, serving as a potential diagnostic marker. The study involved 49 patients and utilized advanced MRI techniques to analyze temporal changes. Despite some limitations, such as a small sample size and single-center design, the findings underscore the importance of imaging in differentiating MS from NMOSD, potentially influencing diagnosis and treatment strategies.

These findings underscore the potential of advanced imaging technologies to detect subtle gray matter differences, aiding in the accurate differentiation between MS and NMOSD.

Clinical

SAP Partners

Differences in Gray Matter Structure May Help Distinguish Multiple Sclerosis From NMOSD

Exposure to fine particulate matter (PM2.5) has been linked to worsening outcomes in patients with multiple sclerosis (MS) and neuromyelitis optica spectrum disorder (NMOSD), according to a recently published study that analyzed data from 2013 to 2022 in Northern Thailand. Published in 

, researchers observed heightened disease severity, increased need for second-line therapies, and prolonged hospitalizations following air pollution exposure.

The study, which included 126 MS and NMOSD exacerbations analyzed for PM2.5 exposure and 135 for PM10 exposure, revealed that PM2.5 significantly worsened clinical outcomes. Patients in the PM2.5 exposure group had higher rates of unfavorable Expanded Disability Status Scale (EDSS) scores at exacerbation (56.3% vs. 23.6%; 

 <.001), along with increased gadolinium enhancement (56.3% vs. 36.4%; 

= 0.03) and greater reliance on plasma exchange therapy (21.1% vs. 7.3%; 

"The findings suggest that PM2.5 exposure exacerbates MS and NMOSD by increasing disease severity, radiological activity (e.g., gadolinium enhancement), and the need for more intensive interventions like plasma exchange. This highlights the neuroinflammatory and neurodegenerative effects of PM2.5, which aligns with its ability to trigger systemic and CNS inflammation. The impact on EDSS scores at exacerbation and 90 days underlines the potential for PM2.5 to contribute to long-term disability," ” lead author Chutithep Teekaput, MD, an assistant professor in the department of internal medicine at Chiang Mai University in Thailand, told 

From a clinical perspective, the exacerbation patterns in the study—peaking 2–4 months after air pollution events—imply a delayed effect of PM2.5 exposure on neuroinflammatory processes. Observations of increased relapses during pollution-heavy seasons might further support this linkage."

All told, patients aged 18 years or older who were diagnosed with MS or NMOSD according to respective clinical criteria, suspected of experiencing a clinical exacerbation, and admitted to the hospital were included in this analysis.

 The primary outcome assessed was the EDSS score during exacerbation. Secondary outcomes included clinical measures, such as the change in EDSS before and after the exacerbation. Baseline data collected during initial exacerbations included patient demographics, medical history, disease status, laboratory findings, and neuroimaging results. Treatment outcomes evaluated included the use of intravenous methylprednisolone, plasma exchange, and the length of hospital stays.

Initiation of Maintenance Therapy After First Attack Improves Long-Term Relapse Risk, Disease Trajectory in MOGAD

Additional findings showed that the need for intensive treatments, such as plasma exchange, was notably higher in patients exposed to PM2.5. Moreover, these patients faced prolonged hospitalizations and more severe disease exacerbations compared with those without air pollution exposure. Authors noted that the radiological findings further corroborated the heightened impact of PM2.5 on disease severity.

Overall, these findings underscored the significant health impacts of PM2.5, which not only increased disease severity but also posed a risk of prolonged hospitalization and permanent disability. Notably, these effects were not observed with PM10 exposure. Authors noted that the highest incidence of exacerbations occurred 4 months after peak air pollution levels, which indicated a delayed but pronounced effect of fine PM.

“The study’s reliance on EDSS provides a robust measure of disability, but incorporating other tools like functional MRI or biomarkers (e.g., cytokine profiles) could enhance the understanding of subclinical neuroinflammation and the broader impact of air pollution on the CNS,” Teekaput noted in the interview. "The increased use of plasma exchange and prolonged hospital stays in PM2.5-exposed groups suggest a need for earlier intervention and closer monitoring of at-risk patients during pollution seasons. Steroid resistance and severe exacerbations might necessitate proactive adjustments in treatment protocols."

According to the authors, this study had several limitations. Air pollution data relied on ground-station measurements, highlighting the need for satellite-based data to enhance coverage and insights. The study population included significantly fewer MS cases compared to NMOSD, differing from distributions seen in Western populations. Prospective data collection may be needed for more robust trend analysis and associations. Furthermore, the study focused only on short-term PM2.5 and PM10 exposure, leaving long-term effects and the impact of other pollutants, such as sulfur dioxide, ozone, and nitrous gases, to be explored in future research.

"Given the findings, integrating air quality monitoring into clinical guidelines and promoting patient education on pollution avoidance are critical. Policymakers should address sources of PM2.5 (e.g., seasonal crop burning) and develop targeted interventions, especially in pollution-prone regions," Teekaput explained to 

REFERENCES
1. Teekaput C, Rachbundit C, Wantaneeyawong C, Teekaput K, Thiankhaw K. Impact of air pollution on the clinical exacerbation of central demyelinating disease: A 10-year data from the Northern Thailand MS and NMOSD registry. 

. Published online January 10, 2025. doi:10.1016/j.msard.2025.106266
2. Thompson AJ, Banwell BL, Barkhof F, et al. Diagnosis of multiple sclerosis: 2017 revisions of the McDonald criteria. Lancet Neurol. 2018;17(2):162-173. doi:10.1016/S1474-4422(17)30470-2
3. Wingerchuk DM, Banwell B, Bennett JL, et al. International consensus diagnostic criteria for neuromyelitis optica spectrum disorders. Neurology. 2015;85(2):177-189. doi:10.1212/WNL.0000000000001729

Exposure to fine particulate matter (PM2.5) is linked to worsened outcomes in multiple sclerosis (MS) and neuromyelitis optica spectrum disorder (NMOSD) patients, as evidenced by a study in Northern Thailand. The study found increased disease severity, higher disability scores, and prolonged hospitalizations associated with PM2.5 exposure. The research highlights the need for national policies to address air pollution sources and calls for further investigation into the mechanisms linking air pollution to central demyelinating diseases. Limitations include reliance on ground-station data and a focus on short-term exposure.

A new study suggests particulate matter exposure exacerbated the severity of multiple sclerosis and neuromyelitis optica spectrum disorder, with significant clinical and radiological impacts.

A newly published study showed that pediatric patients from Black and low-income backgrounds with neuromyelitis optica spectrum disorder (NMOSD) experience significantly worse health outcomes compared with their nonHispanic White and more affluent peers. Published in 

, these findings highlight the interplay of race, socioeconomic factors, and healthcare disparities in managing this rare autoimmune condition of the optic nerve and spinal cord.

Among 38 cases of aquaporin-4-positive (AQP4+) NMOSD diagnosed in pediatric patients, researchers observed that Black or African American (AA) patients had an average Expanded Disability Status Scale (EDSS) score of 2.46 compared with 0.33 in nonHispanic White patients (

 = 0.003). Notably, this minority pediatric patient population also had 2.37 more hospital admissions 

 = 0.002) and spent 28.40 additional inpatient days 

 = 0.002) within 2 years of their diagnosis in comparison with their nonHispanic White counterparts.

“Higher attack severity and mortality have been reported in Black/African American adults with NMOSD, and our study corroborates that similar disparities likely exist in children. Historically, these disparate clinical outcomes were attributed to presumed genetic factors, which disregards the fact that race and ethnicity are social constructs," lead author Kelsey E. Poisson, MD, codirector of the Neuroimmunology Program at Nationwide Children’s told 

 "Black/African American children in our study had a higher degree of socioeconomic disadvantage, which may contribute to poorer outcomes. Given the trend in our study toward more delayed diagnosis in Black/African American children, a more severe burden of disease may develop, requiring longer inpatient hospitalization and ultimately resulting in higher disability."

In this study, researchers analyzed the cases of pediatric patients diagnosed with AQP4+ NMOSD based on 2015 international consensus criteria from 3 tertiary pediatric care centers in the United States between 2009 and 2021.

 Using socioeconomic data linked to the patients' addresses, the investigators assessed the influence of race, ethnicity, and socioeconomic status on disease progression and healthcare utilization over a 2-year period post-diagnosis. Authors noted that the data collection included clinical measures, hospitalization records, and neighborhood-level indicators from data in the U.S. Census.

Cerebral Lesions Considered Important Predictor of Cognitive Deficits in MOGAD

Additional findings showed that pediatric patients with AQP4+ NMOSD who had public insurance experienced a higher relapse rate than those on private insurance (

 = 0.046). Results also revealed that a significantly higher EDSS correlated with pediatric patients who lived in census tracts with a lower median income, higher deprivation index, and higher proportion of population on assisted income, in poverty, and with vacant housing (all, 

< 0.05) at 2 years and at the most recent follow-up. Thus, these results emphasize the role of social determinants in exacerbating health disparities beyond initial diagnosis.

"These neighborhood characteristics illustrate the daily living conditions of our patients and are also influenced by the inequitable distribution of resources through structural racism," Poisson, who also serves as an assistant professor of Clinical Pediatrics and Neurology at The Ohio State University College of Medicine, said. "Living in more disadvantaged neighborhoods can affect health outcomes through environmental exposures, poor access to transportation and healthy food, lack of adequate health insurance and medication adherence, and lower health literacy."

The current study faced several limitations including the exclusion of patients without 2-year follow-up data, which might have made the results biased toward those with better access to care. Furthermore, incomplete documentation may have prevented accurate assessment of delays in initiating disease-modifying therapy. Health care utilization data was also limited for patients seeking care outside study centers, and the sample was less representative of White patients because of regional recruitment biases. Additionally, the assessed neighborhood-level socioeconomic data might not fully capture individual or household-level disparities.

“Ultimately we need larger studies that enable us to pursue sub-analyses on identified disparities in minoritized populations to determine the influence of social determinants of health within these sub-populations,"  Poisson said. "Given that this will be very difficult due to the rarity of AQP4+ NMOSD in children, we simultaneously need to be screening to identify those that are at-risk and offering interventions such as care navigation services, transportation assistance, legal advocacy for unsafe housing, and further caregiver education to promote health literacy about NMOSD."

REFERENCES
1. Poisson KE, Nguyen L, Horn PS, et al. Racial, Ethnic, and Socioeconomic Disparities in Pediatric Aquaporin-4-Positive Neuromyelitis Optica Spectrum Disorder. Pediatr Neurol. Published online October 11, 2024. doi:10.1016/j.pediatrneurol.2024.09.028
2. Wingerchuk DM, Banwell B, Bennett JL, et al. International consensus diagnostic criteria for neuromyelitis optica spectrum disorders. Neurology. 2015;85(2):177-189. doi:10.1212/WNL.0000000000001729

A recent study highlights significant health disparities in pediatric patients with aquaporin-4-positive neuromyelitis optica spectrum disorder (NMOSD), particularly affecting Black and low-income children. These patients experience worse outcomes, including higher disability scores, more hospital admissions, and longer inpatient stays compared to their nonHispanic White peers. Socioeconomic factors, such as public insurance and living in disadvantaged neighborhoods, exacerbate these disparities. The study underscores the need for larger research efforts and targeted interventions to address social determinants of health and improve outcomes for at-risk populations.

A new study revealed stark racial and socioeconomic disparities in pediatric neuromyelitis optica spectrum disorder outcomes, underscoring the need for targeted interventions.

Race, Socioeconomic Status Play Major Role in Disparities Seen in Pediatric NMOSD Outcomes

According to a new announcement, the FDA has approved AbbVie’s foscarbidopa/foslevodopa, marketed as Vyalev, as the first and only subcutaneous 24-hour infusion of levodopa-based therapy for the treatment of motor fluctuations in adults with advanced Parkinson disease (PD).

The company noted that timing for a patient's access to the therapy is dependent on their individual insurance plan, with coverage for Medicare patients expected in the second half of 2025.

“Overall, there is great excitement around the approval of Vyalev. While there are numerous options for the symptomatic treatment of PD, the options become limited as the disease progresses, and patients experience motor fluctuations despite their current treatment," Pavnit Kukreja, PharmD, senior director, neuroscience, US Medical Affairs, AbbVie, told 

 "Previously, only more invasive options, such as surgery, existed. Vyalev offers the ability to continuously deliver the gold standard, levodopa, without surgery, over the morning, day, and night."

The approval was supported by data from the phase 3 M15-736 study (NCT04380142) that assessed the efficacy of continuous subcutaneous infusion of foscarbidopa/foslevodopa, also known as ABBV-951, among adult patients with advanced PD compared with oral immediate-release carbidopa/levodopa (CD/LD IR), along with a 52-week, open-label M15-741 study (NCT03781167) which investigated the long-term safety and efficacy of the treatment. Results from the M15-736 study revealed that patients who received foscarbidopa/foslevodopa reported superior improvement in motor fluctuations, with increased ON time without troublesome dyskinesia and decreased OFF time, compared with oral CD/LD IR.

"The phase 3 study demonstrated superior efficacy over oral IR CD/LD with regards to improvement in On time without troublesome dyskinesia, and the adverse events were mostly mild to moderate and nonserious. We are committed to improving the lives of people impacted by PD and transforming the standard of care with this first-of-its-kind therapeutic option," Kukreja added. 

AbbVie submitted its new drug application (NDA) for ABBV-951

, with M15-736 serving as the basis for the submission.

 The study included 141 adult participants who were randomized 1:1 to either ABBV-951 solution (n = 74) as a continuous infusion under the skin plus oral placebo capsules for CD/LD or ABBV-951 as a subcutaneous infusion plus oral capsules containing CD/LD encapsulated tablets (n = 67).

"For too long, the Parkinson's community has had limited treatment options for advanced disease. Due to the progressive nature of the disease, oral medications are eventually no longer as effective at motor symptom control and surgical treatment may be required," 

, professor of neurology and director of the Parkinson's and Movement Disorder Center at the University of South Florida, said in a statement.

 "This new, non-surgical regimen provides continuous delivery of levodopa morning, day and night."

Led by Hauser, the double-blind, double-dummy, active-controlled study included a screening period (6-60 days), an oral CD/LD stabilization period (2-3 weeks), a double-blind treatment period (12 weeks) for a total of 13 scheduled visits. Eligible participants were at least 30 years old with a diagnosis of idiopathic and levodopa-responsive PD and had inadequately controlled motor fluctuations with an average OFF time of at least 2.5 h/day over 3 consecutive days.

ABBV-951 also showed a good safety profile in the phase 3 study, with most of the reported adverse events (AEs) either mild or moderate in a nature.

 Serious AEs occurred in 8% ABBV-951 group compared with 6% in the CD/LD group. Notably, there as 1 death recorded in the study, believed to be from a treatment-emergent AE while in the CD/LD group. At week 12, ABBV-951-treated patients showed a decrease of 2.75 hours in OFF time, with changes observed as early as the first week. In comparison, those in the oral CD/LD group demonstrated decreases of 0.96 hours during the same stretch (

 for its investigational 24-hour continuous foscarbidopa/foslevodopa pump. In the CRL, the agency requested additional information on the pump as part of its review, but did not request AbbVie to conduct additional efficacy and safety trials related to the drug.

 of foslevodopa/foscarbidopa (Produodopa), the first and only 24-hour infusion of levodopa-based therapy for patients with advanced PD more than a year after it received approval from the European Commission.

 Prior to the launch, the therapy had received authorization through the Decentralized Procedure in the third quarter of 2022, followed by a CE mark in November of 2023. 

The launch was supported by 3 studies: the phase 3 M15-741 study, the double-blind, active-controlled M15-736 study, and a phase 1 pharmacokinetic comparability study. Published in the 

 treated patients in M15-741 demonstrated a mean improvement of 3.8 (SD, 3.3) hours of ON time without dyskinesia and –3.5 (SD, 3.1) hours of normalized OFF time while on the therapy over a 52-week period. Additional, at that time, the percentage of patients experiencing morning akinesia dropped from 77.7% to 27.8%.

 (CRL) to the company's NDA for ABBV-951.

 In the CRL, the agency cited observations identified during inspection of a third-party manufacturer listed in the NDA. The inspection at the facility did not involve ABBV-951 or any of the company's medicine. The CRL did not identify any issues in terms of safety, efficacy, or labeling of ABBV-951, including the device. Furthermore, the letter did not request that the company perform additional efficacy and safety studies related to the drug or device-related testing.

"People living with advanced Parkinson's disease experience daily challenges as a result of uncertainty in managing motor fluctuations, especially as their disease progresses," Roopal Thakkar, MD, executive vice president, research & development, and chief scientific officer, AbbVie, said in a statement.

 "We are proud to bring this innovation to patients who may benefit from motor symptom control through continuous 24-hour administration of Vyalev."

REFERENCES
1. U.S. FDA Approves VYALEV™ (foscarbidopa and foslevodopa) for Adults Living with Advanced Parkinson's Disease. News Release. AbbVie. Published October 17, 2024. Accessed October 17, 2024. https://news.abbvie.com/2024-10-17-U-S-FDA-Approves-VYALEV-TM-foscarbidopa-and-foslevodopa-for-Adults-Living-with-Advanced-Parkinsons-Disease
2. AbbVie submits new drug application to US FDA for investigational ABBV-951 (foscarbidopa/foslevodopa) for the treatment of advanced Parkinson disease. News release. AbbVie. May 20, 2022. Accessed October 17, 2024. https://www.prnewswire.com/news-releases/abbvie-submits-new-drug-application-to-us-fda-for-investigational-abbv-951-foscarbidopafoslevodopa-for-the-treatment-of-advanced-parkinsons-disease-301551779.html?tc=eml_cleartime
3. Soileau MJ, Aldred J, Budur K, et al. Safety and efficacy of continous subcutaneous foslevodopa-foscarbidopa in patients with advanced Parkinson’s disease: a randomized, double-blind, active-controlled, phase 3 trial. 

 2022;21(12):1099-1109. doi:10.1016/S1474-4422(22)00400-8.
4. AbbVie provides regulatory update on ABBV-951 (foscarbidopa/foslevodopa) new drug application. News release. AbbVie. March 22, 2023. Accessed October 17, 2024. https://news.abbvie.com/article_display.cfm?article_id=12567
5. AbbVie launches PRODUODOPA (foslevodopa/foscarbidopa) for people living with advanced Parkinson disease in the European Union. News release. January 9, 2024. Accessed October 17, 2024. https://www.prnewswire.com/news-releases/abbvie-launches-produodopa-foslevodopafoscarbidopa-for-people-living-with-advanced-parkinsons-disease-in-the-european-union-302028989.html
6. Aldred J, Freire-Alvarez E, Amelin AV, et al. Continuous subcutaneous foslevodopa/foscarbidopa in Parkinson’s disease: safety and efficacy results from a 12-month, single-arm, open-label, phase 3 study. 

 2023;12:1937-1958
7. AbbVie Provides U.S. Regulatory Update on ABBV-951 (Foscarbidopa/Foslevodopa). News Release. AbbVie. Published June 25, 2024. Accessed October 17, 2024. https://news.abbvie.com/2024-06-25-AbbVie-Provides-U-S-Regulatory-Update-on-ABBV-951-Foscarbidopa-Foslevodopa

The FDA has approved Vyalev, a subcutaneous 24-hour infusion therapy for advanced Parkinson's disease, offering a new non-surgical treatment option. Supported by phase 3 study data, Vyalev demonstrated superior improvement in motor fluctuations compared to oral carbidopa/levodopa. The therapy provides continuous levodopa delivery, addressing the limitations of oral medications in advanced stages. Most adverse reactions were mild or moderate, with infusion site events, hallucinations, and dyskinesia being the most common. Medicare coverage is anticipated in the latter half of 2025, subject to individual insurance plans.

In clinical trials, patients with Parkinson disease who were treated with foscarbidopa/foslevodopa experienced superior improvement in ON time without dyskinesia compared with oral immediate-release carbidopa/levodopa.

FDA Approves AbbVie’s 24-Hour Foscarbidopa/Foslevodopa Pump for Advanced Parkinson Disease Treatment

A new multi-ethnic cohort study using novel ancestry-informed regression identified novel population-specific risk alleles associated with multiple sclerosis (MS) and successfully fine-mapped previously reported MS loci. These results suggest that novel ancestry-specific genetic variants impact the risk of MS, underscoring the advantage of a trans-ethnic approach for assisting with treatment decisions and prevention of MS in diverse ancestral populations.

All told, data of genome-wide genotypes were available for 4313 Hispanics patients (MS cases, n = 2201; controls, n = 2112) and 3085 African American patients (MS cases, n = 1584; controls, n = 1501). Investigators identified a novel genome-wide significant locus on chromosome 13q14.2 among alleles residing on an African haplotype. For alleles residing on a Native American haplotype, researchers identified a genome-wide significant signal within a known MS locus on chromosome 1p35.2.

"We believe this research, with novel approaches and analytical strategies like these, will help us to further elucidate the underlying molecular pathways involved in MS, which we hope in turn will pave the way for new targeted treatments for MS in all patient populations," lead author Jacob McCauley, PhD, professor at the University of Miami Miller School of Medicine, told 

2024 European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress

, September 18-20, in Copenhagen, Denmark, researcher computed local ancestry with RFMixV2 and conducted genome-wide association with Tractor to acquire ancestry-specific effect-size estimates for alleles from an African, European, and Native American origin after TopMed imputation. Authors then performed a fixed-effects meta-analysis for the ancestry-specific tracts with summary statistics from a European cohort (MS cases, n = ~15K; controls, n = ~27K). Additionally, investigators utilized SuSieX for trans-ancestry fine-mapped in regions that showcased genome-wide significance.

Early Monoclonal Antibody Therapy Reduces Long-Term Disability in Pediatric Multiple Sclerosis

Researchers identified 2, when fine-mapping, of 95% credible sets that represented 2 distinct causal signals at 1p35.2. The first one identified was specific to Native American alleles, the top variant being missense mutation rs145088108, and the second one identified was specific to European alleles, the top variant being the previously identified rs10914539. Notably, investigators achieved 7 additional MS loci with fine-mapping of high resolution. 

These preliminary findings are some of the first to highlight the utility of a trans-ethnic approach to genetic variant discovery for MS risk and genetic fine-mapping efforts that benefited from the inclusion of diverse populations with shorter linkage disequilibrium blocks," McCauley said. "We anticipated that this work would provide novel insights, by leveraging the genetically complex ancestral background of these populations and complement the genetic studies that thus far have only been possible within the larger European ancestry analyses."

A large-scale study presented at ECTRIMS 2024, conducted by the Alliance for Research in Hispanic MS Consortium, identified ancestry-specific genetic factors that influence the risk of developing multiple sclerosis.

Thorsten Mikoteit, MD

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