Results 241 to 250 of about 357,046 (292)

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

open access: yesAnnals 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   +5 more
wiley   +1 more source

Plasma EV Proteomics Identifies ECM Remodeling and Inflammatory Proteins LUM and C7 as Candidate Biomarkers in FSHD

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Facioscapulohumeral muscular dystrophy (FSHD) is one of the most debilitating and common muscular dystrophies. Despite its severity, no approved therapy exists for FSHD patients. However, several therapeutic candidates are currently under development, and some have recently entered clinical trials, marking the need for reliable ...
Mustafa Bilal Bayazit   +11 more
wiley   +1 more source

Eating Experiences of Patients With Cancer in the Hospital - A Qualitative Study. [PDF]

open access: yesJ Hum Nutr Diet
van Elst JM   +6 more
europepmc   +1 more source

Finding the most promising indications for novel treatments in oncology. [PDF]

open access: yesNPJ Precis Oncol
Eckhoff M   +16 more
europepmc   +1 more source

Opioid prescribing practices in the U.S. and subsequent falls: a scoping review protocol. [PDF]

open access: yesSyst Rev
Lovato VI   +5 more
europepmc   +1 more source

Medicare Insurance Type and Broad Genomic Profiling in Metastatic Cancer.

open access: yesJAMA Netw Open
Chow RD   +9 more
europepmc   +1 more source

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