Isabella Ciccone, MPH

NS-050/NCNP-03, an exon skipping investigational therapy, has been cleared by the FDA for the initiation of a phase 1/2 clinical study for the treatment of Duchenne muscular dystrophy.

Boris 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.

Positive 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.

Following 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.

Investigators 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.

Sharon 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.

Phase 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.

Research 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.

As part of our monthly clinician spotlight, NeurologyLive® highlighted sleep disorders expert John Saito, MD, FAAP, FCCP, a pediatric pulmonologist at the Children’s Hospital of Orange County (CHOC).

A 26-year-old man who presented narcolepsy symptoms tested negative for the condition, but a reassessment of the cerebrospinal fluid hypocretin-1 4 years later showed a dramatic decrease.

Maria 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.

Results 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.

Based on a collection of data from clinical trials in Alzheimer disease, the best way to represent and translate the findings to meaningful benefits is through the use of multiple frameworks.

A slowing of disease progression by 30% would result in clearly meaningful differences in patients with mild cognitive impairment and mild dementia.

Caution is warranted for older patients with Duchenne muscular dystrophy who are receiving gene therapy, according to details of a patient death in a single patient trial.

Li-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.

Chaoran 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.

With the recent approval of lecanemab (Leqembi; Eisai), NeurologyLive took a closer look at the Alzheimer pipeline, and the potential agents clinicians should keep an eye on in the coming years.

According 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.

The highest proportions of patients with severe ocular impairment at baseline showed greater improvements in symptom severity on ravulizumab in comparison with placebo after 26 weeks.

Nearly 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.

Patients with generalized myasthenia gravis treated with efgartigimod consistently exceeded treatment compared with placebo regardless of gender in age, disease duration, BMI, and thymectomy.

Howard Fillit, MD, the cofounder and chief science officer of the Alzheimer's Drug Discovery Foundation, shared his reactions to the recent approval of lecanemab and how it opens doors for future drug development in Alzheimer disease.

Medicare recipients living with Alzheimer disease who have documented evidence of beta-amyloid plaque can now receive coverage for lecanemab through participating clinics and registries.

Findings from data on patients with various autoimmune disorders who used remibrutinib, a BTK inhibitor in development for multiple sclerosis, reported no safety concerns in laboratory analyses.

In the phase 3 OPTIMUM study of ponesimod, findings showed that MRI features in patients with multiple sclerosis were significantly associated with clinical scales.

Robert Glanzman, MD, the chief medical officer at the ImmunoBrain Checkpoint, talked about IBC-Ab002 as a promising anti-PD-L1 monoclonal antibody to improve cognition in patients with Alzheimer disease.

Vera Bril, MD, FRCPC, lead investigator of the MycarinG study, shared her reactions to recent approval of rozanolixizumab-noli, the first approved therapy for both subtypes of generalized myasthenia gravis in adults.

Reasons behind the CRL were not related to efficacy and do not impact Amneal’s 2023 financial guidance, which did not include IPX203 revenues.

A group of experts in the care of patients with neurological conditions—Amynah Pradhan, PhD; Katherine Podraza, MD, PhD; Elizabeth K. Seng, PhD; Olivia Begasse de Dhaem, MD, FAHS; Sait Ashina, MD, FAHS—shared their perspectives on hot topics of treatment and management in headache/migraine from the 2023 American Headache Society Annual Scientific Meeting.