Isabella Ciccone, Assistant Editor, NeurologyLive®, has been with the team since September 2022. Follow her on Twitter @iciccone7 or email her at iciccone@mjhlifesciences.com
First Patient Dosed in Phase 2 ACTIVATE Trial of BIA 28-6156 for Parkinson Disease
August 14th 2023The phase 2 clinical trial ACTIVATE investigating BIA 28-6156 for Parkinson disease is currently screening for patients in North America and with the Europe-based study beginning sometime during the third quarter of 2023.
Significantly Larger Hypothalamic Volume in Narcolepsy Type 1 Following H1N1 Infection
August 11th 2023Prior research shows that a significant loss of the hypocretin-producing neurons, an increase in the histaminergic neurons, and contradicting signs of gliosis in the hypothalamus among patients with narcolepsy type 1.
Dosing Commenced in Phase 3 FORTIFY of Limb-Girdle Muscular Dystrophy Type 2i
August 10th 2023BridgeBio Pharma met with the FDA to discuss the use of glycosylated α-dystroglycan levels as a surrogate end point for its phase 3 trial FORTIFY investigating BBP-418 and believes there is the potential for an accelerated approval.
Alzheimer's Association International Conference 2023: Top Expert Interviews
August 5th 2023A group of experts in the care of patients with neurological conditions—Kumar B. Rajan, PhD; Christina Jensen-Dahm, MD, PhD; Nicole Fowler, PhD; Nicholas Ashton, PhD; Jazmyn Muhammad, BS—shared their perspectives on hot topics of treatment and management in Alzheimer disease from the 2023 Alzheimer's Association International Conference.
Innovative Epigenome Editing Adds to Precision Medicine Approach for Alzheimer Disease
August 3rd 2023Boris Kantor, PhD, associate research professor of neurobiology, and Ornit Chiba-Falek, PhD, professor in neurology, both faculty at Duke University, discussed research on an innovative epigenome therapy targeting the APOE gene, a significant genetic risk factor for Alzheimer disease.
Phase 2 Trial Shows Increased Frataxin Levels in Friedreich Ataxia Following CT-1601 Treatment
August 3rd 2023Positive data from the 25 mg cohort study exploring Larimar Therapeutics’ CTI-1601 has been submitted to the FDA, with a meeting scheduled with the agency later this quarter to discuss steps for a phase 2 trial.
Second Patient Dosing Recommended for TSHA-102 Gene Therapy Trial in Rett Syndrome
August 2nd 2023Following a successful first patient treatment experience with TSHA-102, an adeno-associated virus vector-based gene therapy for Rett syndrome, Taysha Gene Therapies was recommended to continue with investigating the therapy in a second patient.
Ropinirole Demonstrates Safety and Efficacy in ALS Progression in 6-Month Trial
August 2nd 2023Investigators concluded that the growing and testing of motor neurons from patient-derived induced pluripotent stem cells could be clinically used for the prediction of ropinirole’s efficacy as a treatment for patients with ALS.
The Potential of AD101 for Managing Symptoms of Alzheimer Disease
August 1st 2023Sharon L. Rogers, PhD, chief executive officer at AmyriAD Therapeutics, discussed AD101, a novel drug that targets the core symptoms in Alzheimer disease, and highlighted the importance of symptom management strategies.
UB-312 Displays Target Engagement of Toxic Alpha-Synuclein in Phase 1 Trial for Parkinson Disease
August 1st 2023Phase 1 trial results investigating UB-312 revealed target engagement of aggregated alpha-synuclein in cerebrospinal fluid of patients with Parkinson disease, providing validation of the vaccine technology to selectively target aggregated, toxic forms of neurodegenerative proteins.
Epigenetic Therapy Demonstrates Efficacy in APOE Reduction for Alzheimer Disease
July 30th 2023Research shows that carrying the APOE e4 variant significantly increases lifetime risk for late-onset Alzheimer disease, and additional evidence suggests that lowering the variant expression may be a promising therapeutic target for the disease.
Integrating Blood-Based Biomarkers as a Revised Approach to Diagnosing Alzheimer Disease
July 27th 2023Maria Carrillo, PhD, chief science officer for the Alzheimer's Association, talked about the release of the updated diagnostic guidelines for Alzheimer disease, which incorporate plasma-based biomarkers.
High Prevalence of CAA-Related Lesions in APOE ε4/4 Homozygotes With Early Alzheimer disease
July 26th 2023Results suggest that the high burden of cerebral amyloid angiopathy-related lesions most likely underlies the 30%-60% incidence of amyloid-related imaging abnormalities in APOE e4/4 carriers treated with approved amyloid therapies.
Diversifying Research and Improving Asian Representation With New Cohort Study for Alzheimer Disease
July 22nd 2023Li-San Wang, PhD, the Peter C. Nowell, MD, professor of pathology and laboratory medicine, Perelman School of Medicine at the University of Pennsylvania, discussed a study that aims to address the underrepresentation of Asian populations in Alzheimer disease research.
Insights on Uncovering the Gut-Brain Interaction and Cognitive Function in Alzheimer Disease
July 19th 2023Chaoran Ma, MD, PhD, assistant professor at University of Massachusetts Amherst, talked about the association between bowel movement frequency, the gut microbiome, and cognitive function in patients living with dementia.
Caregivers Perceived Reciprocity Reduces Behavioral Symptoms in Patients With Alzheimer Disease
July 16th 2023According to diary data, caregiver's perceived reciprocity showed a substantial direct influence on the occurrence of behavioral symptoms on both the current day and the subsequent day for patients with Alzheimer disease and related dementias.
Zilucoplan Demonstrates Consistent Efficacy in Phase 3 RAISE Study of Generalized Myasthenia Gravis
July 12th 2023Nearly half of patients treated with zilucoplan were considered responders on Myasthenia Gravis Activities of Daily Living (MG-ADL) at the first week, suggesting a rapid onset of efficacy.