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
Eculizumab Generates High Response Rates in Generalized Myasthenia Gravis
By the end of the open-label extension, 15-17% of patients showed clinically meaningful first response with eculizumab treatment.
Zolgensma Shows Benefits in SMA Type 1
Patients with spinal muscular atrophy maintained the ability to thrive and achieved motor milestones previously unseen in natural history studies.
No-Charge Genetic Testing Partnership for Muscular Dystrophy Community Announced
In addition to no-charge testing, patients may have access to the Detect Muscular Dystrophy program, a counseling program designed to aid health decisions and follow-up care.
Intrathecal Zolgensma Shows Early Efficacy in SMA
Nearly all patients in this cohort achieved a clinically meaningful >3-point increase during the study period, demonstrating a consistent response to the gene therapy.
Edaravone Effective Regardless of Forced Vital Capacity in ALS
No unexpected safety signals were reported in the post hoc analysis.
Vazegepant Meets Primary End Points in Migraine Pain Freedom
After meeting co-primary end points of pain freedom and freedom from the most bothersome symptom, the acute migraine agent has been cleared for a phase 3 trial by the FDA.
Viltolarsen Available Through Expanded Access Program
Eligible patients with Duchenne muscular dystrophy may receive the drug while it is under priority review by the FDA.
Cannabis Helps Improve Spasticity in Multiple Sclerosis
Patients with multiple sclerosis who reported cannabis use noted that it was helpful in relieving pain, as well as other symptoms, such as sleep, depression, anxiety, and/or stress.
Rituximab Superior in Reducing Relapse Risk for NMOSD Patients
The risk of relapse was higher with treatment of mycophenolate mofetil in patients with neuromyelitis optica spectrum disorder and other first-line immunosuppressants, compared to treatment with rituximab.
Topiramate Oral Solution ET-101 Shows Bioequivalence With Topamax
Eton Pharmaceuticals plans to submit a new drug application for ET-101 in the third quarter of 2020, anticipating its formulation to be the first approved oral topiramate solution.
New Guidelines for Huntington Disease Include Physical Therapy Interventions
A combination of different physical therapy interventions designed to improve fitness, motor function, and gait are all recommended when guiding clinical practice for patients with Huntington disease.
Multiple System Atrophy Drug Verdiperstat Wins Fast Track Designation from FDA
The ongoing phase 3 trial of the Biohaven drug is expected to be completed October 20, 2021.
Stress-Related Disorders Linked to Higher Risk of Neurodegenerative Disease
Statistically significant associations between stress-related disorders were identified in those with Alzheimer disease, but not with Parkinson disease or amyotrophic lateral sclerosis.
Triptan Refills for Migraine Treatment Decrease Over Time
Patients opted for nontriptan acute medications such as opioids and nonsteroidal anti-inflammatory drugs instead of staying on triptans at 12- and 24-month follow-ups.
High-Dose Biotin Fails in Phase 3 for Progressive Multiple Sclerosis
Further results of the SPI2 trial will be presented at the upcoming American Academy of Neurology 2020 Annual Meeting in Toronto.
FDA Approves MRI Labeling for NeuroPace RNS System
The label expansion now allows patients who require non-brain MRI monitoring to receive treatment with the stimulation device.
Antiseizure Medication Tolerability Still a Significant Hurdle
Despite the introduction of second-generation ASMs, drug tolerability has been relatively unchanged over the last 30 years.
NDA for Qtrypta Accepted for Acute Migraine
The drug’s Prescription Drug User Fee Act date is set for October 20, 2020.
Stroke Unit Touted Most Effective to Treat Hemorrhage
Compared with intensive care units or normal wards, stroke units were associated with reduced mortality in patients with intracranial hemorrhage.
Cognitive Impairment Worsens During Multiple Sclerosis Relapse
Patients with relapsing multiple sclerosis showed declining scores on tests of cognitive processing speeds from baseline to 3-month follow-up post-relapse.
Clene Banks on Novel Gold Nanotherapeutic for ALS and Beyond
The chief executive officer of Clene Nanomedicine discussed CNM-Au8, their novel investigational drug that will be used in the world’s first platform trial of potential ALS treatments.
Low-Dose Cannabidiol Effective for Reducing Convulsive Seizures in Dravet Syndrome
Both the 10- and 20-mg doses were associated with clinically relevant reductions in convulsive seizures with a favorable safety and tolerability profile.
Cladribine Effectively Reduces Risk of Secondary Progressive Multiple Sclerosis
Similar effects were observed for each proxy SMPS component when compared with placebo.
Endovascular Therapy Displays Similar Outcomes to Intravenous Thrombolysis
When performed by itself, endovascular therapy had greater rates of intracranial hemorrhage compared to a combination of both endovascular therapy and intravenous thrombolysis.
Diazepam Nasal Spray Treatment Now Available for Prescription
The drug was previously approved for the treatment of acute repetitive seizures and granted 7 years of orphan drug exclusivity in January 2020.
Phase 3 Trial of Gaboxadol Is Underway for Angelman Syndrome
Gaboxadol is the first treatment in 50 years associated with positive change in outcomes in Angelman syndrome.
FDA Approves Eptinezumab, Ofatumumab Accepted for Supplemental Biologics License Application, Deutetrabenazine Fails in Tourette Syndrome
Neurology News Network for the week ending February 29, 2020.
Cure Rare Disease Seeks to Lead Innovation in Rare Disease Treatment
The president and founder of Cure Rare Disease discussed the company’s custom therapeutics for patients with rare diseases, including the use of CRISPR gene-editing technology to develop treatments for Duchenne muscular dystrophy.
Earlier Ocrelizumab Treatment in MS Has Positive Long-term Impact
Compared with patients who initially received interferon treatment, those given ocrelizumab had superior disability progression after a 6-year follow-up.
Siponimod Significantly Slows MS Disability Progression in Patients With Shorter Disease Duration
Patients with a disease duration of
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