Abstract: Methods for alleviating symptoms in a Smith Kingsmore Syndrome (SKS) patient using low doses of a mTOR inhibitor. Also provided herein are methods to determine suitable doses of a mTOR inhibitor for a SKS patient to alleviate at least one symptom associated with SKS with little or no negative impact on behavior features such as the sleep pattern of the SKS patient.

Abstract: A system for objectively analyzing medical image data for the presence of diffuse white matter abnormalities (DWMA) is configured to identify and determine DWMA characteristics that are not visually apparent. As compared to subjective visual diagnosis, objectively determined DWMA characteristics may be automatically compared to each other, and may be compared to and associated with various scales, evaluations, or other assessment criteria used to measure aspects of infant development. As a result, the disclosed system may objectively determine an impact that objectively determined DWMA characteristics will have on one or more developmental scales, which can be expressed as a time deficit, score deficit, or other value indicative of development deficits.

Abstract: A medicinal dosage device may comprise a base having dosing indicators operably connected to the base. The dosing indicators may comprise about four arms spaced at about ninety degree intervals, which may indicate a three hour block of time. The dosing indicators may be selectably movable as a unit relative to the base to indicate medicinal dosage times for at least two different medications.

Abstract: Various aspects of the disclosure relate to a tracheal implant comprising (a) a trachea processed to comprise one or more incisions providing a helical configuration to the processed trachea; and (b) a subject-specific synthetic support.

Abstract: Provided herein is a method of diagnosing and treating a subject suffering from atopic dermatitis (AD), the method comprising: (a) obtaining a skin biopsy from a subject suspected of suffering from AD; (b) determining a level of RNA expression in the skin biopsy of genes selected from the 89ADGES gene panel; (c) comparing the determined level of RNA expression of the selected genes to the level of RNA expression of the selected genes in a reference sample comprising RNA expression products from normal healthy skin cells; (d) diagnosing the subject as suffering from AD when specific genes are up-regulated compared to the reference sample and when specific genes are down-regulated compared to the reference sample; and (e) treating the subject with a therapy effective for the treatment of AD. Methods of managing treatment of a subject suffering from AD are also provided.

Abstract: A method for determining a personalized full dose of a melphalan compound (e.g., melphalan) in a reduced intensity conditioning regimen (RIC) prior to hematopoietic cell transplantation for a subject based on pharmacokinetic features of the melphalan compound administered to the subject at a test dose.

Abstract: A method for motion correction of Magnetic Resonance (MR) images is provided. The method includes acquiring a k-space dataset for an object using an MR scanner, detecting or identifying corrupted k-space data from the acquired k-space dataset, extracting the corrupted k-space data from the acquired k-space dataset, recovering the corrupted k-space data, combining uncorrupted k-space data of the acquired k-space dataset with the recovered k-space data to form a full k-space dataset, and reconstructing an image for the object based on the full k-space dataset. A magnetic resonance imaging system for correcting corrupted k-space data of an entire k-space dataset is also provided.

Abstract: Described herein are prodrugs of cysteamine and pharmaceutically acceptable salts, solvates, and esters thereof. Also described herein are pharmaceutical compositions comprising prodrugs of cysteamine, or pharmaceutically acceptable salts, solvates, and esters thereof, and methods of treatment comprising administering prodrugs of cysteamine, or pharmaceutically acceptable salts, solvates, and esters thereof.

Abstract: An augmented neuromuscular training system and method for providing feedback to a user in order to reduce movement deficits associated with injury risk, prior injury or disease pathology.

Abstract: Some embodiments of the invention include methods for treating an animal for cystic fibrosis comprising one or more administrations of one or more compositions comprising saracatinib. Other embodiments of the invention include treating an animal N for cystic fibrosis comprising one or more administrations of one or more compositions comprising saracatinib, optionally a corrector of ?F508 CFTR, and optionally a potentiator of ?F508 CFTR. Still other embodiments of the invention include methods for treating a human with cystic fibrosis caused by the F508 deletion mutation in the cystic fibrosis transmembrane conductance regulator (CFTR), comprising one or more administrations of one or more compositions comprising saracatinib, and optionally VX770, VX809, or both. Additional embodiments of the invention are also discussed herein.

Abstract: Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increasing a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention also relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia or ?-thalassemia.

Abstract: A ventricular catheter assembly including a proximal catheter and a cooperating cranial cover. The cranial cover includes a base plate having an opening aligned with a burr hole in the skull of a person. A guide extends upwardly from the base plate and receives the proximal catheter.

Abstract: Disclosed are compositions and methods for the reduction of C5a mediated immune inflammation. The methods, in various aspects, may include the step of administering a C5aR antagonist to a subject in need of such treatment. In one aspect, the subject in need may have a lysosomal acid storage disease. Therapeutic kits and articles of manufacture are also disclosed.

Abstract: A method of inhibiting inflammation with milk oligosaccharides or glycoconjugates containing the oligosaccharides.

Abstract: Disclosed herein are methods for treating an individual having a Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) infection. The method may comprise administering a JAK inhibitor, for example ruxolitinib (JAKAFI®), to an individual in need thereof, such individual generally being an individual having, or suspecting of having, SARS-CoV-2 infection. The individual in need thereof may be an individual having, or suspected of having or at risk for developing SARS-CoV-2 infection-related cytokine storm. The individual in need thereof may further be an individual having, or suspected of having SARS-CoV-2 infection-related pneumonia.

Abstract: Provided herein are 2?-fucosyllactose compounds and methods of using such for treating inflammatory bowel diseases (IBD) (e.g., Crohn's disease (CD) or ulcerative colitis (UC)) or alleviating or reducing the risk of relapse in IBD.

Abstract: Disclosed are methods of alleviating or preventing one or more symptoms associated with fragile X syndrome in an individual in need thereof via administration of a therapeutically effective amount of a GABA(A) alpha 2 and/or 3 partial agonist. The one or more symptoms may include impaired functional communication, anxiety, inattention, hyperactivity, sensory reactivity, autonomic nervous system dysregulation, aberrant eye gaze, self-injury, aggression, seizures, EEG abnormalities, including but not limited to, abnormal spectral analysis, event related potentials which may include auditory and visual responses, abnormalities in cortical responses as evoked by transcranial magnetic stimulation including resting and active motor thresholds and abnormal responses in measures of cortical inhibition and excitation, aberrant impaired cognitive function, compromised daily living skills, or a combination thereof.

Abstract: One or more techniques and/or systems are disclosed for a myringotomy tube including a tube body having a first end and a second end. The tube body may comprise a shaft operably connecting the first end and the second end. The first end may comprise a first visual element. The second end may comprise a second visual element. The first visual element may be different than the second visual element. The myringotomy tube may be a single piece. The myringotomy tube may be multiple pieces.

Abstract: Methods and compositions disclosed herein generally relate to methods of identifying, validating, and measuring clinically relevant, quantifiable biomarkers of diagnostic and therapeutic responses for blood, vascular, cardiac, and respiratory tract dysfunction, particularly as those responses relate to septic shock in pediatric patients. In particular, the invention relates to identifying two or more biomarkers associated with septic shock in pediatric patients, obtaining a sample from a pediatric patient having at least one indication of septic shock, then quantifying from the sample an amount of two or more of said biomarkers, wherein the level of said biomarker correlates with a predicted outcome.

Abstract: Disclosed are methods of treating an individual at risk for a negative outcome associated with a treatment for a disease, in particular a proliferative disorder such as cancer. The method may include the steps of creating a predictive assay that includes both a biomarker and a genetic mutation. The predictive assay indicates the likelihood of a negative outcome associated with a particular treatment in a particular individual, such that an individual may be administered a treatment less likely to be associated with a negative outcome.