Results 251 to 260 of about 705,460 (317)

Unraveling 4‐Phenylbutyrate's Therapeutic Role in SLC6A1 Disorders: Pharmacochaperoning Over HDAC Inhibition

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Variants in SLC6A1, encoding the GABA transporter 1 (GAT‐1), cause epilepsy, autism spectrum disorder, and developmental delay via loss of GABA uptake, impaired trafficking, and ER retention. We previously found that 4‐Phenylbutyrate (PBA), an FDA‐approved drug, restores GABA uptake and reduces seizures in SLC6A1‐related disorders ...
Melissa B. DeLeeuw, Karishma Randhave, Ekta Anand, Ziang (Debbie) Song, Wangzhen Shen, Jing‐Qiong Kang   +5 more
wiley   +1 more source

Reply to: Rethinking intra-arrest thrombolysis: the role of patient selection and timing. [PDF]

Resusc Plus
Ohshimo S, Couper K, Böttiger BW, Dehghani S, Scquizzato T, Grunau B.   +5 more
europepmc   +1 more source

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito, Kamal Benguerba, Ken Tsuchida, Kazushige Yazawa, Isao Tsumiyama, Hiromitsu Kayama, Sandra P. Reyna, Farid Khan, Richard S. Finkel   +8 more
wiley   +1 more source

Incidence and Outcomes of in-Hospital Cardiac Arrest and Cardiopulmonary Resuscitation in the Kidney Replacement Therapy Population: Protocol for a Linked National Disease Registry Study. [PDF]

JMIR Res Protoc
McLaren R, Hole B, Tuharska Z, Pippias M.   +3 more
europepmc   +1 more source

Alisertib Induces Cell Cycle Arrest and Autophagy and Suppresses Epithelial-to-Mesenchymal Transition Involving PI3K/Akt/mTOR and Sirtuin 1-Mediated signaling pathways in Human Pancreatic Cancer Cells [Retraction]. [PDF]

Drug Des Devel Ther
europepmc   +1 more source

Intravoxel incoherent motion MRI for early detection and assessment of renal injury after cardiac arrest and resuscitation in a rat model. [PDF]

Eur Radiol Exp
Zhang J, Zhan N, Ye H, Liu S, Tan Y, Zhang J, Huang T, Wu Z, Yang Z, Liu Z.   +9 more
europepmc   +1 more source

Scoping review of contemporary adult venoarterial extracorporeal membrane oxygenation (VA ECMO) selection criteria and decision-making mechanisms. [PDF]

J Extra Corpor Technol
Johal AK.
europepmc   +1 more source

Barriers to action: behavioral determinants of bystander intervention in medical emergencies - a scoping review. [PDF]

BMC Emerg Med
Hassan S, Jabbar ABA, Raza S, Bheesham S, Demetres M, Shafiq Y, Razzak J.   +6 more
europepmc   +1 more source