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
NeuroVoices: Crystal Proud, MD, on Improving SMA Outcomes Through a Combination Approach
The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, provided perspective on the phase 4 RESPOND study assessing nusinersen in individuals with SMA who already received gene therapy.
Ketogenic Agent Tricaprilin Shows Potential Benefit in Pilot Study of Migraine
At month 2 of a 3-month treatment period, investigators observed an improvement of 2.75 days favoring tricaprilin.
Stromal Cell Therapy NurOwn Shows Significant Impact on Neurofilament Light, Markers of Neuroprotection
Accounting for baseline disease covariates, NurOwn-treated participants had reduced neurofilament light values from baseline to week 20 compared with placebo (P <.05).
AI Model SCORE-AI Shows Clinical Utility in Reading Abnormal EEGs, Distinguishing Seizure Types
Interrater agreement between SCORE-AI and human experts was notable, with almost perfect agreement for generalized epileptiform abnormalities and substantial agreement for focal epileptiform discharges, among other findings.
Increased Levels of Alpha-Linolenic Acid Linked With Disease Progression in ALS
Higher levels of the n-3 fatty acid eicosapentaenoic acid and the n-6 fatty acid linoleic acid were associated with a lower risk of death during follow-up.
Migraine Preventive Atogepant Effective in Individuals With Prior Medication Failures
In a cohort of patients who experienced 4-14 migraine days/month, treatment with atogepant continued to improve patients’ migraine status regardless of prior medication failures.
Cell Therapy CAP-1002 Continues to Show Preservation of Cardiac Function in Open-Label Extension Study of DMD
Building on positive 1-year data, 24-month data showed statistically significant differences in the Performance of the Upper Limb scale in treated patients in the open-label extension relative to those on placebo in the double-blind period.
Nusinersen Shows Additive Effects in Spinal Muscular Atrophy After Zolgensma Administration
On average, younger and older participants who were unable to sit without support improved from baseline on CHOP-INTEND scores over a 183-day period.
Diroximel Fumarate Maintains Efficacy in Elderly Populations of Multiple Sclerosis
Over a 96-week period, more older patients with relapsing multiple sclerosis on diroximel fumarate achieved no evidence of disease activity-3 status vs younger patients.
FDA Grants Traditional Approval to Lecanemab as Therapy for Early-Stage Alzheimer Disease
The decision was based on findings from the phase 3 confirmatory Clarity AD study, in which treatment with the agent resulted in significant reductions in amyloid-ß in the brain.
Immunoglobulin Therapy Hyqvia Continues to Show Positive Results on Chronic Inflammatory Demyelinating Polyneuropathy
Patients on Hyqvia experienced longer time to relapse compared with those on placebo, with Kaplan-Meier estimate curves separating early, at approximately week 4.
Exploring FDA-Approved Treatments for NMOSD to Manage MOGAD: The METEOROID Study
METEOROID is the first study to evaluate the efficacy and safety of satralizumab, an FDA-approved therapy for NMOSD, in patients with MOGAD.
Stem Cell Therapy BRT-DA01 Demonstrates Positive Safety, Feasibility in Early-Stage Study of Parkinson Disease
At 1 year, treatment with the investigational cell therapy was well tolerated with no major safety issues and showed evidence of cell survival and engraftment.
Burel Goodin, PhD, on Building Trust in Underserved Communities to Improve Clinical Trial Outcomes
The professor of anesthesiology at Washington University in St. Louis discussed the importance of effectively communicating and reciprocating with underserved communities that are of research interest.
Increased Mortality Risk and Shorter Life Expectancy Associated With Sleeping Pills
Among sleeping pill nonusers, when compared with patients who got a medium amount of sleep, extremely short or long sleepers had approximately 30% higher risk of all-cause mortality and 3-5 years shorter life expectancy at age 30-50 years.
FDA Issues Complete Response Letter to Amneal for Parkinson Agent IPX203
Reasons behind the CRL were not related to efficacy and do not impact Amneal’s 2023 financial guidance, which did not include IPX203 revenues.
CT1812 Shows Positive Impact on Underlying Brain Function in Alzheimer Study
Through a quantitative electroencephalogram, investigators observed brainwave changes in several regions of the brain following treatment with CT1812.
Episode 92: Highlights From the 2023 CMSC, SLEEP, AHS, and ATMRD Annual Meetings
Mind Moments®, a podcast from NeurologyLive®, brings you exclusive interviews with Amit Bar-Or, MD; Gavin Giovannoni, MBBCh, PhD; Dayna Johnson, PhD, MPH, MSW, MS; John Winkelman, MD, PhD; Laxman Bahroo, DO; Ling Wan-Albert, OTD, OTR/L; Sara Pavitt, MD; and Ali Ezzati, MD. [LISTEN TIME: 28 minutes]
NeuroVoices: Burel Goodin, PhD, on Improving Pain Medicine Care in Underserved Populations
The professor of anesthesiology at Washington University in St. Louis provided insight on his presentation at AHS 2023 discussing the need to engage underserved populations.
FDA Clears Roche’s Beta-Amyloid and Total-Tau Assays for Alzheimer Assessment
With the approval, Roche has 3 FDA-cleared tests that are reflective of the main Alzheimer disease pathologies and help clinicians more completely define the disease biologically.
Gaining a Better Understanding of Idiopathic Hypersomnia, Uses of Low-Sodium Oxybate
Marisa Whalen, PharmD, associate director of Medical Affairs at Jazz Pharmaceuticals, discussed presentations at SLEEP 2023 on the real-world burdens of idiopathic hypersomnia and the smooth transition to low sodium oxybate in patients with narcolepsy.
Rozanolixizumab Becomes First FDA-Approved Therapy for AChR and MuSK Antibody Positive Generalized Myasthenia Gravis
In a phase 3 study, rozanolixizumab demonstrated statistically significant differences vs placebo on primary and secondary outcomes over a 43-day treatment period.
Prophylactic Lumbar Drainage Following Aneurysmal Subarachnoid Hemorrhage Shows Additional Benefits
At 6 months, a larger proportion of patients on standard of care experienced unfavorable outcomes, as demonstrated by modified Rankin Scale scores greater than 3, in comparison with lumbar drainage.
Switching to Ocrelizumab Following Natalizumab Associated With Lowest Relapse, Discontinuation Rates
In addition to increased relapse rates, use of fingolimod was associated with a 49% higher risk for disability accumulation in comparison with ocrelizumab.
Gene Therapy AMT-130 Shows Promising Results on Several Outcomes in Phase 1/2 Study of Huntington Disease
Following the positive results, uniQure expects to initiate a third cohort in the ongoing trial to further investigate both low and high doses of AMT-130 in combination with perioperative immunosuppression.
Biohaven Submits New Drug Application for Troriluzole as Spinocerebellar Ataxia Type 3 Therapy
In a phase 3 study, a subgroup of patients with spinocerebellar ataxia treated with troriluzole demonstrated a numerical treatment benefit relative to placebo on the primary end point of change in f-SARA scores.
Pegcetacoplan to be Discontinued in ALS Following Disappointing Phase 2 Results
In a trial of 250 adults with sporadic ALS, pegcetacoplan failed to meet its primary end point of change in Combined Assessment of Function and Survival rank scores relative to placebo after a 52-week stretch.
CD40L Inhibitor Frexalimab Demonstrates Significant Reduction of Gadolinium-Enhancing Lesions in Multiple Sclerosis
High and low doses of frexalimab resulted in reductions of new or enlarging T2 lesions and total T1 gadolinium-enhancing lesions over a 3-month period.
Special Episode: SRP-9001 Approved As First Gene Therapy for Duchenne Muscular Dystrophy
Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Natalie Goedeker, CPNP. [LISTEN TIME: 12 minutes]
Biomarkers Predictive of NMOSD Disability Reduced Through Inebilizumab
Compared with placebo, inebilizumab-treated participants showed attenuated biomarker elevation during attacks and reduced biomarker levels over time in the absence of adjudicated attacks.
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