
FORTIS (NCT04174105) is a first-in-human study, enrolling 4 participants with late-onset Pompe disease to assess the Astellas Pharma therapy.
FORTIS (NCT04174105) is a first-in-human study, enrolling 4 participants with late-onset Pompe disease to assess the Astellas Pharma therapy.
Having recently been awarded a grant from the Michael J. Fox Foundation, Mary Hamby, PhD, VP of research at Cognition Therapeutics, and Lisa Ricciardi, the company’s CEO, sat down to discuss the potential disease-modifying effects for this patient population.
The chief scientific officer of FibroBiologics discussed the benefits of fibroblast cell technology for this patient population, particularly the absence of adverse effects. [WATCH TIME: 3 minutes]
Relative to other PD-related genetically associated groups, only those with LRRK2-associated PD demonstrated a significantly increased risk of stroke compared with controls.
The cognitive neurologist at the University of California San Francisco discussed the need to continue research on why patients develop cognitive changes following COVID-19 infection and how clinicians can care for them. [WATCH TIME: 4 minutes]
In previous phase 1 settings, CY6463 has demonstrated an impact on biomarkers relevant to neurodegeneration and cognitive impairment.
This was the first study to demonstrate a preventive treatment signal in migraine when an approved oral CGRP receptor antagonist is dosed as needed for the acute treatment of migraine.
In addition to those with MS, the approval also indicates the formulation for the treatment of patients with spinal cord injuries and other spinal cord diseases.
Frail people or those with lower cognitive reserve were the only variables that benefitted from higher doses, whereas APOE status and education had no significant interaction.
The chief scientific officer of FibroBiologics spoke on fibroblast cell technology and its specific benefits in comparison stem cell therapy. [WATCH TIME: 4 minutes]
At the 2-year time point, more than 40% of preadolescent and approximately 55% of adolescent patients retained treatment with perampanel after initiation during routine clinical care.
Investigators concluded that the prompt was ‘well-suited’ for use in clinical care, but additional research is needed to better understand the association between PELHS-QOL-2-Medications and race.
The senior director of patient management, care, and rehabilitation at the National Multiple Sclerosis Society discussed the steps needed to take to improve relatively new wellness strategies. [WATCH TIME: 3 minutes]
Investigators noted that while epilepsy did not increase the risk for sleep disorders, there was a strong association identified with TSC-association neuropsychiatric disorders, necessitating early detection.
After changes were made to the AVATAR phase 3 study in Rett syndrome, Anavex provided context on the timing of the adjustments, which were approved by regulatory bodies in the UK and Australia, where the trial was conducted.
Neurology News Network for the week ending February 5, 2022. [WATCH TIME: 4 minutes]
Matthew Klein, MD, MS, FACS, the COO of PTC Therapeutics, discussed a supplemental new drug application for the treatment in a younger group of patients, which has been granted priority review by the FDA.
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending February 4, 2022.
The biotech company announced a collaboration with NS Pharma around CAP-1002’s commercialization and distribution ahead of its HOPE-3 phase 3 trial in Duchenne muscular dystrophy.
In the RAISE trial, patients treated with zilucoplan exhibited clinically meaningful and statistically significant improvement from baseline in Myasthenia Gravis-Activities of Daily Living Profile total score at week 12, when compared with those who received placebo.
The assistant clinical professor at Vanderbilt University School of Medicine discussed ways of incorporating and encouraging women physicians to enter the neurology specialty. [WATCH TIME: 4 minutes]
Most child neurologist respondents were generally early in their career, had diverse subspeciality training, and tended to work at large, resource-rich centers capable of multimodal neuromonitoring.
Although research illustrates the persistence of inequities between women and men in neurology, 3 women leaders shared encouraging thoughts and experiences highlighting the progress made in recent years.
Collaboration can result in greater success than what one can achieve alone, and for women physicians, this rings truer than ever before, writes Jill M. Farmer, DO, MPH.
In light of National Women Physicians Day, the assistant clinical professor at Vanderbilt University School of Medicine commented on the roles women have played historically, and her perspective on where they stand now. [WATCH TIME: 12 minutes]
Theresa Sevilis, DO, writes on picking a career path in medicine as a woman, and how deciding to jump ship from a traditional path was among the best decisions she's made.
The assistant clinical professor at Vanderbilt University School of Medicine shared what National Women Physicians Day means to her, and the history behind its celebration. [WATCH TIME: 6 minutes]
For women, the decision to opt for an unconventional career path can be inspired by conflicting responsibilities and balancing life with work. But, as Sana Syed, MD, MPH, explains, following your passion is still an achievable dream despite these challenges.
Although findings suggest better efficacy of consyntropin when compared with vigabatrin, the randomized trial was underpowered due to incomplete enrollment.