Victoria Johnson, Assistant Editor for GeneTherapyLive, joined the MJH Life Sciences team in October 2020, originally writing for NeurologyLive. Follow her on Twitter @VictoriaJNeuro or email her at vjohnson@genetherapylive.com.
Donanemab Shows Slowing of Decline in Alzheimer Phase 2 TRAILBLAZER-ALZ Study
The follow-up TRAILBLAZER-EXT and broader population TRAILBLAZER-ALZ 2 studies are currently ongoing.
Third Ventricle Width: An Accessible Biomarker to Differentiate Between PD and PSP
Andrea Quattrone, MD, discusses how his team worked to create a simpler biomarker after developing MRPI 2.0.
Erenumab Improves Patient-Reported Outcomes in Refractory Migraine
Erenumab also demonstrated improvements in standard measures of efficacy, such as monthly migraine days.
Hypertensive Disorders of Pregnancy Associated With Worse Cognition
Women with HDP were found to perform worse on cognitive tests 15 years after delivery than their counterparts.
Higher Levels of Physical Activity Linked With Less MS Fatigue
Neurological disability, as assessed by EDSS, was also found to be associated with higher levels of MS fatigue.
Out-of-Pocket Costs Steadily Increasing for Neurologic Care
A recent analysis of UnitedHealthcare insurance claims revealed increases of almost 200% for some services between 2001 and 2016.
Nilotinib Shows No Beneficial Effect in Patients with Parkinson
Researchers from Northwestern University found nilotinib had an acceptable safety profile, but lacked efficacy that would encourage further study in patients with Parkinson disease.
Athira Pharma Awarded $15 Million Grant to Investigate ATH-1017 for Alzheimer
Both ongoing ACT-AD and LIFT-AD studies are assessing Athira’s investigational therapy ATH-1017.
SOD1 Variants Show Significant Association with ALS Risk
Researchers from the University of Turin also found that an NEFH variant showed a protective association with ALS.
MOVE-FA Study Initiated for Friedriech’s Ataxia in Children and Young Adults
PTC Therapeutics announced the initiation of its third study of 2020 investigating vatiquinone.
Moderate to Vigorous Physical Activity Pivotal in Managing Cardiovascular Risk Post-Stroke
Annual reviews of moderate to vigorous physical activity may help patients maintain adequate levels of activity after stroke.
QOD Rimegepant Shows Slight but Significant Reduction in Migraine Days
More patients on rimegepant than placebo reached at least a 50% reduction in monthly migraine days.
LAMS and RACE Scales Lead Predictive Accuracy of Symptomatic Large Anterior Vessel Occlusion
All 7 predictive scales analyzed were found to be highly accurate, with some more feasible than others.
Larger Infarct Volume, Younger Age Associated With Poorer Arterial Ischemic Stroke Outcomes
The associations, while modest, were not strong enough to be accurately used as predictors of outcome, but the findings offer value for pediatric care.
King’s and Milano-Torino Staging Systems Effective in Assessing ALS Clinical Progression
Results from the post-hoc analysis, funded by Mitsubishi Tanabe Pharma America, were recently announced.
Initial Results Prove Positive for Low-Dose AXO-AAV-GM1 in GM1 Gangliosidosis
These data follow a recent announcement from Sio Gene Therapies that the first patient in the high-dose cohort has been dosed.
Transdermal CBD Efficacious in Children With Developmental and Epileptic Encephalopathies
Efficacy was observed in patients with DEEs such as West Syndrome, Lennox-Gastaut syndrome, and Dravet syndrome.
Oral Anticoagulation for AFib Insufficient After Ischemic Stroke in Patients with SVD
Patients with any presence of small vessel disease had a 2-times greater risk of recurrent ischemic stroke than those without.
CNM-Au8 Improves MUNIX(4) Score in Amyotrophic Lateral Sclerosis
The novel gold nanocrystal suspension has also been shown to remyelinate chronic MS lesions.
THC-Containing Cannabinoids: A New Avenue for MS Treatment
Stephen Krieger, MD, discusses a new phase 3 trial of nabiximols, which is now recruiting patients with MS.
Personal KinetiGraph Shows Improvements Compared to Standard Care in Parkinson Disease
MDS-UPDRS score improvements from baseline were significantly greater in patients using the Personal KinetiGraph.
Cannabidiol Maintains Efficacy Long-Term in Tuberous Sclerosis Complex
Seizure reduction was observed to be sustained for up to 192 weeks in the Expanded Access Program data.
Progressive Aerobic Exercise May Help Improve Cognitively Impaired Patients With MS
No cognitive improvements were observed for exercise versus waitlist groups of cognitively normal patients with MS.
Fenfluramine Shows Clinical Benefit in Dravet for Adults as Well as Children
The US Early Access Program for fenfluramine allowed access to the treatment for patients who were ineligible for clinical trials.
Personalized Neuromodulation Therapy Reduces Seizures Safely in Drug-Resistant Epilepsy
Microburst stimulation was also found to be more tolerable than standard stimulation in patients with drug-resistant epilepsy.
Pediatric Epilepsy Laser Ablation Shows Efficacy With Few Complications
The registry is planning to expand, with 5 additional centers recently agreeing to participate and institutional research ethics applications underway.
Intermittent Midazolam Administration Yields Return to Functionality for Seizure Cluster Episodes
Data from this post-hoc analysis show slight differences between repeated administration of 1 and 2 doses of midazolam.
Both Low- and High-Dose Perampanel Effective and Well-Tolerated
The data suggest that some patients with partial-onset seizures may benefit from lower doses to mitigate treatment emergent adverse events, but these must be tailored to each patient.
Real World Experience Study of Brivaracetam for Focal Seizures Announced
Top line results from the retrospective EXPERIENCE study of brivaracetam are expected to read out in early 2021.
Perampanel Reports High Compliance and Good Tolerance in Phase 4
High compliance rates and well-tolerance of perampanel indicate its efficacy as a treatment for partial onset seizures.