Results 121 to 130 of about 72,467 (253)

Listening across frequencies: Capturing seizures of all patients to advance antiseizure medication development

Epilepsia, EarlyView.
Abstract Seizure frequency has been the primary endpoint in epilepsy trials, with enrollment usually requiring ≥4 seizures per month. This threshold is more and more misaligned with clinical reality, as the availability of more treatment options has reduced baseline seizure burden, with a risk of excluding a proportion of patients from trials. Although
Stéphane Auvin, Jacqueline French
wiley   +1 more source

Antisense oligonucleotides reverse SPTLC1-related hereditary sensory neuropathy in a mouse model. [PDF]

Brain
Meng J, Ma S, Lone MA, Lam HW, Zhang Q, Cheng S, Mackie S, Graham E, Kedzior H, Demetriou C, Ziak N, Andreoli L, Beggs S, Koch S, Clark AJ, Bennett DL, Hornemann T, Muntoni F, Reilly MM, Zhou H.   +19 more
europepmc   +1 more source

Compensatory rearrangement of parvalbumin interneuron voltage‐gated sodium channel subunits in a mouse model of Dravet syndrome

Epilepsia, EarlyView.
Abstract Heterozygous loss‐of‐function variants in the gene SCN1A, which encodes the voltage‐gated sodium channel (VGSC) pore‐forming (α) subunit NaV1.1, lead to a spectrum of neurological disease, including Dravet syndrome. NaV1.1 is prominently expressed at the proximal portion of the axon initial segment (AIS) of fast‐spiking γ‐aminobutyric ...
Ania K. Dabrowski, Ala Somarowthu, Sophie R. Liebergall, Damaris N. Lorenzo, Ethan M. Goldberg   +4 more
wiley   +1 more source

Dysregulation of the DNA damage response by phosphorothioate antisense oligonucleotides. [PDF]

Nat Commun
Hjelmgren L, Zhou Q, Schmidli S, Gloudemans M, Czapik T, Roudi S, Honcharenko M, Hagey DW, El Andaloussi S, Farnebo M.   +9 more
europepmc   +1 more source

Progress report on new epilepsy treatments: A summary of the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVIII). II. Treatments in more advanced clinical development

Epilepsia, EarlyView.
Abstract This article summarizes data for 13 investigational treatments for which at least preliminary seizure outcome data in patients with epilepsy were reported at the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices held in Madrid, Spain, on May 3–6, 2026.
Meir Bialer, Cecilie Johannessen Landmark, Matthias J. Koepp, Emilio Perucca, Piero Perucca, Torbjörn Tomson, H. Steve White, Elaine Wirrell   +7 more
wiley   +1 more source

Antisense Oligonucleotides: Technological Advances, Clinical Progress, and Expanding Therapeutic Frontiers. [PDF]

Pharmaceutics
Xu L, Zhang H, Jiang B, Jiang Y, Lu H.
europepmc   +1 more source

Progress report on new epilepsy treatments: A summary of the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVIII). I. Treatments in preclinical and early clinical development

Epilepsia, EarlyView.
Abstract Over the last 34 years, the Eilat Conference on New Antiepileptic Drugs and Devices has provided an interactive forum for stakeholders to discuss investigational and recently licensed treatments for seizures and epilepsy. The Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVIII) took place in Madrid, Spain, on May 3 ...
Meir Bialer, Cecilie Johannessen Landmark, Matthias J. Koepp, Emilio Perucca, Piero Perucca, Torbjörn Tomson, H. Steve White, Elaine Wirrell   +7 more
wiley   +1 more source

Targeting TLK2 with antisense oligonucleotides as a new strategy in acute myeloid leukemia. [PDF]

Front Oncol
Lin HY, Khou S, Lind E, de Oliveira Rebola KG, Dean A, Zhang H, Tolentino A, Maazi H, Revenko A, Agarwal A.   +9 more
europepmc   +1 more source

A prospective natural history study protocol for clinical trial readiness in synaptic disorders

Epilepsia, EarlyView.
Abstract Objective STXBP1‐related disorder (STXBP1‐RD) and SYNGAP1‐related disorder (SYNGAP1‐RD) are two common genetic synaptopathies that are associated with epilepsy, developmental delay, intellectual developmental disorder, and behavioral problems.
Jillian L. McKee, Sarah M. Ruggiero, Kristin Cunningham, JoeyLynn Coyne, Ian McSalley, Michael C. Kaufman, Bintou Bane, Torrey Chisari, Jonathan Toib, Carlyn Glatts, Sarah Tefft, Julie M. Orlando, Viveknarayanan Padmanabhan, Alexander K. Gonzalez, Alicia Harrison, Charlene Woo, Stephanie A. Zbikowski, Rency Dhaduk, Johanna Mercurio, Macie McCarthy, Jan H. Magielski, Zachary Grinspan, Megan Abbott, Juliet Knowles, Hsiao‐Tuan Chao, Katherine Xiong, Elizabeth Berry‐Kravis, Sepideh Tabarestani, J. Michael Graglia, Kathryn Helde, Virginie McNamar, Charlene Son Rigby, James Goss, Scott Demarest, Andrea Miele, Benjamin Prosser, Michael J. Boland, Samuel R. Pierce, Ingo Helbig   +38 more
wiley   +1 more source

ClinASO: An open-source platform for rapid drug discovery of gapmer antisense oligonucleotides. [PDF]

Mol Ther Nucleic Acids
Chen S, Liu H, Cong D, Dong A, Wang Y, Bi J, Guo S, Yang J, Wang X, Ren G, Zhang K, Wang H, Lai F, Dang Y.   +13 more
europepmc   +1 more source