Abstract: A cerebral monitoring device for determining failure of a shunt used to treat pediatric hydrocephalus. The cerebral monitoring device including a controller configured to control an optical instrument to emit multi-spectral light to illuminate a cranial tissue of the patient, and control an optical detector to detect multi-spectral light emitted from the illuminated cranial tissue of the patient. The controller also configured to compare the emitted multi-spectral light to the detected multi-spectral light, compute cerebral blood flow (CBF) data based on the comparison, compute a pulsatility index of the CBF data, compute a pulsatility index of blood pressure of the patient, compute intracranial pressure (ICP) based on the pulsatility index of the CBF data and the pulsatility index of the blood pressure, and determine shunt failure based on blood oxygen saturation of the patient and the ICP of the patient.

Abstract: A power tool, such as a cast saw is disclosed. The cast saw is equipped with at least one thermocouple and and/or at least one infrared detector that measure the temperature of the cutting blade on the cast saw or the area in the cast immediately surrounding the cutting blade. The cast saw is also equipped with at least one LED that can alert a user of the cast saw when blade or the area in the cast immediately surrounding the blade is approaching or has reached a temperature that may burn a patient. Also disclosed is a shroud for the cutting blade of the cast saw. The shroud not only surrounds at least part of the blade but also houses the thermocouple or infrared detector and the LEDs(s). A method of operating the cast saw to cut a cast off of a patient is also disclosed.

Abstract: Compositions and methods for the detection and treatment of ADHD are provided.

Abstract: The neuroblastoma immunopeptidome is enriched with peptides derived from proteins essential for tumorigenesis including the unmutated peptide QYNPIRTTF (SEQ ID NO: 1) discovered on HLA-A*24:02 which is derived from the neuroblastoma dependency gene and master transcriptional regulator PHOX2B. To target QYNPIRTTF, peptide-centric chimeric antigen receptors (PC-CARs) were developed via a counter panning strategy using predicted potentially cross-reactive peptides. Informed by computational modeling, PHOX2B peptide¬ centric CARs were demonstrated to also recognize QYNPIRTTF (SEQ ID NO: 1) presented by HLA-A*23:01 and the highly divergent HLA-B*14:02. Potent and specific killing of neuroblastoma cells expressing these HEAs in vitro was shown along with complete tumor regression in mice.

Abstract: Provided herein are engineered red blood cells expressing rare blood antigen group profiles, and methods of making use the same, are described. Also provided are recombinant reagent red blood cells that express or lack the expression of at least one protein (e.g., a blood group antigen) on its surface and uses thereof.

Abstract: The disclosure is directed to crystalline forms and mixtures thereof of R-fasoracetam. Such crystalline forms include Form II monohydrate R-fasoracetam and the anhydrate form of R-fasoracetam. The disclosure further includes mixtures of the anhydrate form of R-fasoracetam together with one or more of Form I monohydrate R-fasoracetam and Form II monohydrate R-fasoracetam.

Abstract: The present disclosure relates, in part, to lipid nanoparticles (LNPs), and compositions comprising the same, which have increased stability in amniotic fluid, and methods of use thereof for in utero delivery of therapeutic agents, to treat and/or prevent diseases and/or disorders in a fetal subject. The present disclosure further relates to an ex vivo assay for screening LNP stability in a test fluid.

Abstract: Compositions and methods for diagnosing and treating asthma and asthma patients a Th2 high asthma phenotype are disclosed.

Abstract: Compositions and methods for the treatment of ribosomopathies and regeneration of hematopoietic stems cells are provided.

Abstract: A system configured to support growth and development of a premature fetus is disclosed. Specifically, a method and apparatus configured to provide extracorporeal support for premature fetuses is disclosed.

Abstract: Disclosed are systems, methods, and devices for percutaneous retrieval of objects, such as endovascular devices, from an internal body space. The present inventions have vascular, non-vascular (gastrointestinal), and surgical (laproscopic) applications. The inventions include a a wire woven distal end that is formed from wires and includes a substantially cylindrical main body section comprising a leading end, a trailing end, and a middle portion extending between the leading end and the trailing end and defining a longitudinal axis of the main body section, and a flare defining a mouth of the wire woven distal end that projects circumferentially from the leading end of the main body section, wherein when at least a portion of the retrieval end is transitioned to a compressed state following capture of an object, the retrieval end exerts an inward force that at least partially collapses or compresses the object.

Abstract: Compounds and methods for preventing and/or treating one or more symptoms of sickle cell diseases (SCD) by administering at least one of the compounds are provided. The compounds are chemically modified to increase bioavailability and activity, e.g., so that the compounds prevent adhesion and sickling of red blood cells (RBCs).

Abstract: The present invention relates generally to the field of using udenafil or a pharmaceutically acceptable salt thereof in patients who have undergone the Fontan operation.

Abstract: This disclosure is directed to methods of methods of computing a composite measurement comprising employing two or more sub-instruments to measure one or more clinical symptoms of mitochondrial dysfunction or mitochondrial disease. This disclosure is also directed to methods of assessing and managing a subject with mitochondrial dysfunction or mitochondrial disease using a composite measurement.

Abstract: Compositions and methods are provided for the alleviation of pathology induced by traumatic brain injury.

Abstract: Provided herein are combinations of capsaicin and TrpV1 modulators that are useful for treating capsaicin-responsive diseases or disorders.

Abstract: The present disclosure relates to apparatus and methods for safely and securely holding an infant and medical equipment proximal to the infant. In particular embodiments, an apparatus may comprise an infant support device configured to support an infant proximal to a user. Exemplary embodiments may also comprise a medical equipment support device configured to support medical equipment proximal to the infant.

Abstract: Provided herein are compositions and methods for delivering a molecular therapeutic to the cochlea of a subject. The methods comprise administering an adeno-associated virus (AAV) to the cerebrospinal fluid of the subject. The AAVs encode a therapeutic transgene for molecular therapy. Optionally, the therapeutic transgene may be operably linked to a cochlea-specific promoter.

Abstract: Compositions and methods for the treatment of H-ABC leukodystrophy are disclosed.

Abstract: Compositions and methods for treatment of mitochondrial respiratory chain dysfunction and other mitochondrial disorders are provided. Also disclosed are a number of screening assays having utility for the identification of agents which modulate the phenotype associated with mitochondrial respiratory chain dysfunction.