NeurologyLive

12/22/2024

In our latest NeuroVoices, Barry Ticho, MD, PhD, of Stoke Therapeutics, sat down at to discuss the promising data behind STK-001, an investigational antisense oligonucleotide in development for . American Epilepsy Society

📘Read Now: https://www.neurologylive.com/view/neurovoices-barry-ticho-disease-modifying-potential-stk-001-treating-dravet-syndrome

12/22/2024

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on PepGen’s investigational new drug application for its phase 2 CONNECT2-EDO51 study, assessing PGN-EDO51 in patients with , and will issue an official clinical hold letter within 30 days.

As of July 2024, 2 participants in the ongoing CONNECT1 study have received 4 doses of PGN-EDO51 at 10 mg/kg, which has been generally well tolerated, with initial results expected in early 2025.

12/21/2024

Dosing has begun in a new phase 1, proof-of-mechanism study testing the therapeutic effects of QurAlis’ QRL-101, a Kv7 ion channel opener, as a potential treatment for patients with ALS. Topline results from this single-dose, placebo-controlled study are anticipated in the first half of 2025.

The proof-of-mechanism study will evaluate single doses of QRL-101 in approximately 12 patients with ALS, focusing on excitability biomarkers like the strength-duration time constant (SDTC), safety, tolerability, and plasma pharmacokinetics, with topline results expected in early 2025.

12/21/2024

Findings from a cohort of nearly 19,000 people from the Health and Retirement Study (HRS) revealed that obstructive sleep apnea, a common midlife sleep disorder, may be a significant risk factor for dementia onset, with disproportionate impact on women.

The cumulative incidence of dementia was higher in people with OSA, particularly for women, across different age groups.

12/21/2024

In newly announced data from the phase 2b PADOVA study, treatment with investigational prasinezumab (Roche) did not meet its primary end point of time to confirmed motor progression in patients with Parkinson disease; however, the treatment was safe, and showed positive trends on multiple secondary and exploratory end points. Full results from PADOVA are expected to be presented at an upcoming medical meeting.

Although the primary goal was not met, prasinezumab demonstrated encouraging effects on secondary and exploratory measures, such as time to worsening motor function, Clinical Global Impression of Change, MDS-UPDRS motor scores.

12/20/2024

Julie Ziobro, MD, PhD, of University of Michigan; John Schreiber, MD, of Children’s National Hospital, talked about 2 studies presented at that used the Pediatric Epilepsy Research Consortium genetics database to study factors influencing latency in genetic testing and drug-resistant epilepsy. American Epilepsy Society

https://www.neurologylive.com/view/using-perc-database-gather-insights-epilepsy-genetics-julie-ziobro-john-schreiber

12/20/2024

In recent news, the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CervoMed’s oral investigational drug neflamapimod for the treatment of patients with frontotemporal (FTD).

The company noted that it is actively collaborating with clinical experts to plan a proof-of-concept study in FTD but is pausing all preparations for its previously planned phase 3 trial of neflamapimod in early-stage dementia with Lewy bodies until the full analysis is complete.

Most recently reported topline data from the RewinD-LB phase 2b trial in patients with dementia with Lewy bodies showed that neflamapimod failed to meet its primary end point and secondary end points.

12/20/2024

John Michael Schreiber, MD, discusses how genetic testing enhances the diagnosis and management of pediatric epilepsy, emphasizing its importance in tailoring treatment strategies for affected patients. Watch now: bit.ly/3ZNv1Lo

12/20/2024

Edgewise Therapeutics has announced positive topline data from its phase 2 CANYON trial, with results showing that investigational sevasemten, formerly known as EDG-5506, met its primary end point in change of creatine kinase among patients with Becker .

The positive data, along with data from GRAND CANYON anticipated in early 2025, are thought to be part of a potential future authorization submission for sevasemten in Becker.

12/18/2024

Watch the on-demand broadcast to listen to experts discuss how NOURIANZ® (istradefylline) works differently for “off” episodes for patients with PD. bit.ly/47OVUS4 See Prescribing Information for NOURIANZ bit.ly/3KSzp5l

12/18/2024

Innovation in “On” time is here. Watch the on-demand broadcast to listen to Robert A. Hauser, MD, FAAN, Stuart Isaacson, MD, FAAN, and Neepa J. Patel, MD discuss a new treatment option for adult patients with Parkinson’s disease . bit.ly/48SDjG5

12/18/2024

At , pediatric epileptologists at Norton Children's Neuroscience Institute presented a poster study examining factors influencing seizure freedom in patients with perinatal arterial ischemic stroke. To learn more, check out the article on our site now! American Epilepsy Society

Epileptologists Siddharth Jain, MD, MBBS, and Cemal Karakas, MD, provided commentary on a poster presentation at AES 2024 focusing on factors influencing seizure freedom in children who’ve experienced neonatal and perinatal arterial strokes.

12/18/2024

At , Jessica Nickrand, PhD, and Allyson Eyermann, of the Child Neurology Foundation, discussed multidisciplinary collaboration, innovations in care, and the importance of holistic, family-centric approaches to improving patient outcomes. American Epilepsy Society

View Now: https://www.neurologylive.com/view/insights-child-neurology-foundation-advancing-holistic-epilepsy-care-jessica-nickrand-allyson-eyermann

12/18/2024

At , Jacqueline A. French, MD, of NYU Grossman School of Medicine, discussed how innovative startups are using proof-of-concept studies and devices like responsive neurostimulators to improve patient outcomes. American Epilepsy Society

👉 Watch Now: https://www.neurologylive.com/view/advancing-epilepsy-treatment-novel-approaches-predictive-technologies-jacqueline-french

12/16/2024

Donald S. Wood, PhD, president and chief executive officer at the MDA, provided thoughts and perspectives on the promising design and educational attainment for the 2025 MDA Clinical & Scientific conference.

Donald S. Wood, PhD, president and chief executive officer at the MDA, provided thoughts and perspectives on the promising design and educational attainment for the 2025 MDA Clinical & Scientific conference.

12/16/2024

New study finds obesity in MS linked to higher disease activity, worse quality of life, and lower cognitive function. Overweight/obesity tied to increased MRI activity; obesity associated with poorer health, fatigue, and more pain.

More:

Obesity was associated with faster progression of disability, including a higher risk of reaching higher EDSS scores, suggesting that increased body weight contributes to MS severity over time.

12/16/2024

The FDA has granted breakthrough therapy designation to Sanofi’s tolebrutinib, an investigational Bruton’s tyrosine kinase (BTK) inhibitor, for patients with non-relapsing secondary progressive multiple sclerosis.

Details: https://www.neurologylive.com/view/fda-grants-tolebrutinib-breakthrough-therapy-non-relapsing-secondary-progressive-ms

12/15/2024

At , Babitha Haridas, MD, MBBS, of Johns Hopkins Medicine, spoke about the common occurrence of multifactorial causes of (SE), such as a child experiencing vomiting due to a viral illness, that leads to missed medication doses and subsequent SE. American Epilepsy Society

The pediatric epileptologist at Johns Hopkins Medicine discussed common triggers and potential preventive measures of status epilepticus in LGS, as well as the importance of involving families and caregivers. [WATCH TIME: 4 minutes]