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Chronic pain, sleep disorders, neuropathy, and anxiety were the most common conditions that showed improvement in the study.

MediciNova will be moving forward with a phase 2b/3 clinical trial of the ALS therapy, also known as MN-166, in 150 patients. If successful, its data will be used to support an NDA.

Patients with multiple sclerosis who were exposed to teriflunomide during pregnancy experienced similar risks of major birth defects and spontaneous abortions to the general population’s rates of 2% to 4% and 15% to 20%, respectively.

The scale includes 11 observable characteristics of CDM1 and was established through the merging of experiences and advice from patients, caregivers, therapeutic experts, and the FDA.

The Aquestive Therapeutics product, marketed as Exservan, was previously granted an orphan drug designation in January 2018. Its NDA is supported by a number of studies which sought to confirm its bioequivalence to its reference listed drug.

The study also added to a growing base of findings which suggest that patients who have failed preventive medications prior to trial participation have lower placebo responses, which could have implications for clinical trial design.

The senior preclinical and clinical imaging scientist at the National Institute of Neurological Disorders and Stroke spoke about the potential of the central vein sign to help improve the time to an accurate diagnosis in MS.

In an ongoing phase 2 study, 21 nonambulatory participants with Duchenne muscular dystrophy passed the 52-week treatment mark.

Data from the EVOLVE-1 and EVOLVE-2 phase 3 clinical trials suggest that galcanezumab (Emgality, Eli Lilly) can maintain ≥50% responder rates equivalent to that of placebo while reducing more monthly migraine days for 5 months after halting treatment.

Exome-based screenings may allow for maximum diagnostic yield in an increasingly crowded field of genetic tests.

Results of a recent real-world study at 2 MS centers have uncovered misdiagnosis rates of 17% and 19%, with patients facing an average time of misdiagnosis of 4 years during which they may be subject to numerous risks due to improper treatment.

Neurology News Network for the week of April 13, 2019.

A shorter yet deeper therapeutic coma for treatment of refractory status epilepticus may be more effective and safer than the currently recommended therapeutic coma duration of 24 to 48 hours.

Over the course of 2 years, findings revealed that serum NfL levels were significantly higher in patients with evidence of disease activity compared to those with no evidence of disease activity as well as 35 age-matched healthy controls.

The chief scientific officer of the Parkinson’s Foundation spoke about the developing interest in genetic forms of Parkinson, the potential to target therapies in that direction, and how a genetic treatment is the next big stage for the field.

The PEGASUS principal investigator and Translational Neurology Head of the Interdisciplinary Brain Center at Massachusetts General Hospital and Harvard Medical School spoke about the Amylyx Pharmaceuticals product’s potential in Alzheimer.

Pembrolizumab resulted in a decrease in the detection of programmed cell death protein 1 on lymphocytes in both cerebrospinal fluid and peripheral blood, with 5 of the 8 patients displaying clinical improvement or stabilization.

The differential diagnosis of frontotemporal dementia is complicated by its heterogenous clinical presentation, with symptoms that overlap with several other dementias.

Amylyx Pharmaceuticals announced the trial of the 2-drug combination is seeking to enroll 100 patients with late-stage mild cognitive impairment or dementia due to Alzheimer, with plans to complete the study in 2020.

A new analysis provides a comprehensive update on this significant, and growing, cause of disability and death.

The FDA cited 2 deficiencies in the NDA: certain nonclinical studies were not included to allow for assessment of chronic administration of fenfluramine; and an incorrect version of the clinical data was submitted.

The chief scientific officer at the Parkinson's Foundation spoke about the need to increase awareness about the proper care of patients with Parkinson disease.

The planned phase 1/2 trial of the recombinant AAV5 vector treatment, the first one-time administered AAV gene therapy to enter clinical testing for Huntington disease, is expected to begin dosing patients in the second half of 2019.

The senior preclinical and clinical imaging scientist at the National Institute of Neurological Disorders and Stroke gave a presentation on a volumetric segmented echo-planar-imaging (3D-EPI) sequence, which could be used to detect novel biomarkers such as the central vein sign rapidly.

The International Headache Society has issued a number of recommendations for the proper design of trials for the prevention of pediatric migraine, hoping to address the challenges which are unique to pediatric patient populations.

Repeated brain MRI scans show four small gray matter lesions. What’s in your differential?

Data from a national cohort has shown a 48% rate of confirmed symptomatic fractures, which the authors suggested was expected to be a large underestimation of the real frequency in the DMD patient population.

Neurology News Network for the week of April 6, 2019.