FDA Rejects Duchenne Muscular Dystrophy Drug Golodirsen, Citing Safety Concerns
The regulatory agency cited 2 concerns in the complete response letter: the risk of infections related to intravenous infusion ports and renal toxicity.
Pfizer Recalls Relpax for Migraine Due to Potential Microbiological Contamination
Those with an existing inventory of eletriptan hydrobromide 40 mg tablets, lots AR5407 and CD4565, should stop use and distribution, and quarantine immediately.
Phase 3 Trial of Vutrisiran Evaluates Potential Therapy for Hereditary Transthyretin-Mediated Amyloidosis
The investigational candidate is the subject of a phase 3 study assessing efficacy and safety in adults with hATTR amyloidosis with transthyretin mutation.
The Symbiotic Relationship of Artificial Intelligence and Neurology
The associate chief of the MS division and professor of neurology at the University of Pennsylvania described the relationship between artificial intelligence and medicine, and how he sees it evolving in the future.
Jeffrey Cummings, MD, ScD, on Investigational Therapies for Alzheimer Disease
The director emeritus of the Cleveland Clinic Lou Ruvo Center for Brain Health and vice chair of the department of brain health at the University of Nevada Las Vegas spoke about cognitive enhancing agents, disease-modifying therapies, and neuropsychiatric therapies under investigation for Alzheimer disease.
Exploring the Impact of Caregiver Depression on Patients With Dementia
The neurohospitalist and assistant professor of neurology at the University of California San Francisco spoke about how caregiver depression is an important risk factor associated with increased healthcare use among individuals with dementia.
Diazepam Buccal Film Beneficial in Refractory Epilepsy With Seizure Clusters
The new drug application for Libervant is expected to be completed in the fourth quarter of 2019, which if approved, would offer a potentially first in class oral treatment for breakthrough or cluster seizures.
Young Blood Plasma Beneficial in Parkinson Disease
Six-month results show significant improvements in motor symptoms, mentation, behavior, and mood, with a prolonged residual benefit.
FDA Addresses Zolgensma Gene Therapy Data Manipulation
The agency said the gene therapy should remain on the market while it assesses the situation and does not impact their evaluation of data from the human clinical trials.
GRF6019 Shows Positive Phase 2 Results in Mild to Moderate Alzheimer Disease
Subjects receiving GRF6019 maintained cognitive and functional status over a period of 6 months, and dosing was reported to be safe and well-tolerated.
Gene Therapy for CLN6 Batten Disease Shows Positive Interim Results
Interim data from the first 8 pediatric patients showed that the AAV-CLN6 gene therapy demonstrated a positive impact on motor and language function compared to a natural history dataset, as well as in comparison to in-study sibling pairs.
FDA Accepts NDA for ET-105 for Seizure Treatment
The FDA has assigned the application a PDUFA target action date of March 17, 2020.
Early MRI Scans Can Predict Long-Term Disease Outcomes in Relapse-Onset Multiple Sclerosis
Early focal inflammatory disease activity and spinal cord lesions are predictors of long-term disease outcomes, which may be useful in counseling patients and personalizing treatment plans.
Intensive Blood Pressure Lowering Is Likely to Reduce Stroke Recurrence
Despite the fact that the rate of reductions for recurrent stroke in the intensive group were not significant in this study, a meta-analysis of 3 previous trials supports the use of a blood pressure target less than 130/80 mm Hg to reduce stroke recurrence.
Introducing Innovative Technologies Into Stroke Standard of Care
The co-director of the Gates Stroke Center and Cerebrovascular Surgery at Kaleida Health and director of endovascular stroke treatment and research medical director of neuroendovascular services at Gates Vascular Institute spoke about unmet needs in stroke and the value of introducing innovative technologies into the space.
Smartphone-Based Obstructive Sleep Apnea Screening Device Gets FDA OK
The Drowzle algorithm was tested against polysomnography results and provided a sensitivity of 93.7% to detect moderate and severe obstructive sleep apnea.
PMZ-1620 Enters Phase 3 Trial As a Potential Treatment For Cerebral Ischemic Stroke
The investigational therapy has the potential to be a first-in-class neuronal progenitor cell therapeutic with anti-apoptotic activity that improves cerebral blood flow and neurological outcome in stroke.
FDA Approves First Generics of Lyrica
Pregabalin is approved for use in partial onset seizures, as well as postherpetic neuralgia, fibromyalgia, and for the management of neuropathic pain associated with spinal cord injury and diabetic peripheral neuropathy.
FDA Cites Active Ingredient Issue With Riluzole For Treatment of Amyotrophic Lateral Sclerosis
The complete response letter cited issues with an active pharmaceutical ingredient used in the Biohaven 2017 bioequivalence study that was manufactured between 2014 and 2016 in an Apotex facility.
Improving Alzheimer Care in the Emergency Department: Q&A With Manish Shah, MD, MPH
The professor of emergency medicine at the University of Wisconsin-Madison School of Medicine and Public Health spoke about the challenges physicians face in the emergency department when dealing with patients that are cognitively impaired.
Using a Precision-Medicine Platform to Address Polypharmacy in Cognitive Impairment
uMETHOD Health has implemented a precision-medicine platform to create personalized, multidomain care plans for the treatment of dementia and mild Alzheimer disease.
Transcranial Direct Current Stimulation, Working Memory Training Beneficial in Prodromal Alzheimer Disease
While single modality therapy is safe, feasible, and effective, researchers demonstrated that a combined modality shows greater domain-specific cognitive enhancements with higher transferability and sustainability.
CSF Biomarkers of Neurodegeneration May Predict Progression in Mild Cognitive Impairment
Participants with high CSF Aβ1-42/tau and lower NPTX2 levels experienced greater decline throughout the 36 months than all other subgroups on memory acquisition, delayed recall, and CDR-sb.
Staccato Alprazolam Suppresses Epileptic Activity in Phase 2a Study
All doses of Staccato alprazolam reduced the standardized photosensitivity range at 2 minutes; the effect was sustained for the 0.5 mg dose through 4 hours and 6 hours for the 1 mg and 2 mg doses.
Predicting Brain Injury Outcomes in Survivors of Cardiac Arrest: Best Practices From AHA
The group’s goal is for the clinical research community to develop an accurate, precise clinical test to predict brain injury recovery after resuscitation from cardiac arrest.
FDA Accepts New Drug Application for Opicapone as an Adjunctive Treatment for Parkinson Disease
The prescription drug user fee act target date is scheduled for April 26, 2020.
Parkinson Disease Gene Therapy PR001 Granted Fast Track Designation
A phase 1/2 clinical trial is expected to be initiated in the second half of 2019.
Seltorexant Achieves Primary, Secondary Endpoints in Insomnia
The mean decrease from baseline in latency to persistent sleep was 15 minutes for placebo, 30 minutes for seltorexant 5 mg, 50 minutes for 10 mg, and 48 minutes for 20 mg.
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