Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject.

Abstract: In one aspect, the disclosure provides a method of defining a Raman signature of a culture component, the method comprising: obtaining a Raman spectrum of a culture component in a non-interfering or minimally-interfering solution, identifying peaks in the Raman spectrum that are associated with the culture component, obtaining a Raman spectrum of a culture medium comprising the culture component, and, removing peaks of the culture component in the Raman spectrum of the culture medium that are distorted compared to the peaks identified in the Raman spectrum of the culture component in a non-interfering or minimally-interfering solution.

Abstract: The present invention provides compounds and compositions thereof which are useful as inhibitors of Bruton’s tyrosine kinase and which exhibit desirable characteristics for the same.

Abstract: Embodiments related to methods and wearable medical detecting systems for detecting disease states and/or treatment states of a subject are described. In one embodiment, a wearable structure may include one or more radiation detectors use to detect a time varying radiation signal emitted from a labeled compound within a body portion of interest. The radiation signal may be analyzed to determine one or more signal characteristics that may be compared to one or more predetermined standard characteristics associated with known disease and/or treatment states to determine a current disease and/or treatment state of a subject.

Abstract: Pharmaceutical compositions containing anti-beta amyloid (A?) antibodies or AD-binding fragments thereof are provided. These pharmaceutical compositions find use in the treatment of abnormal accumulation or deposition of A? in the central nervous system, mild cognitive impairment, and AD-associated disorders such as Alzheimer's disease.

Abstract: A medical process comprising administering a digital symbol substitution test (DSST) to a patient, administering a trails marking test (TMT) to the patient after the administration of the DSST, and administering a recall test, wherein the recall test includes requesting the patient to recall digits and/or symbols from the DSST.

Abstract: Featured are biornarkers for, e.g., diagnosis and prognosis of spinal muscular atrophy (SMA) as well as identification of responders to treatment of SMA. Also provided are methods of treating subjects with SMA.

Abstract: The invention generally relates to the use of nuclear transport modulators, e.g., CRM1 inhibitors, of Formula (I) for treating epilepsy in a subject. Subjects can have unprovoked seizures without prior warning that affect one or both hemispheres of the brain. The disclosed methods can reduce the severity and occurrence of these unprovoked epileptic seizures and can result in resolution of epilepsy. The method comprises administering to the subject an effective amount of a compound represented by Formula (I) or a pharmaceutically acceptable salt thereof.

Abstract: A medical process comprising: identifying one or more symptoms, determining at least one measurement descriptive of the one or more symptoms, creating a model of the at least one measurement, obtaining the at least one measurement descriptive of the one or more symptoms from at least one symptomless subject, using the model to transform the at least one measurement from the at least one symptomless subject into a reference data, obtaining the at least one measurement descriptive of the one or more symptoms from at least one subject with the one or more symptoms, using the model to transform the at least one measurement from the at least one subject with the one or more symptoms into a patient data, and comparing the reference data to the patient data.

Abstract: Disclosed herein are antisense compounds and methods for decreasing Tau mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Tau-associated diseases, disorders, and conditions.

Abstract: This invention relates to alpha-4 binding antibodies, and fragments thereof.

Abstract: The present invention provides compounds and compositions thereof which are useful as inhibitors of Bruton's tyrosine kinase and which exhibit desirable characteristics for the same.

Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject.

Abstract: Methods of the disclosure may include obtaining a first set of medical images at a first time point and a second set of medical images at a second time point, each set including at least two medical images. First and second algorithms may be used to calculate, respectively, first and third brain volume (BV) values at the first time point based on two or more images from the first set of medical images and second and fourth BV values at the second time point based on two or more images from the second set of medical images. A mathematical weight may be applied to at least one of the first, second, third, or fourth BV values. The first and third BV values may be averaged, and the second and fourth BV values may be averaged to determine overall BV values at the first and second time points, respectively.

Abstract: Methods of activating GDF11 proteins in vitro as well as formulations of mature GDF11 polypeptides with enhanced solubility at neutral pH are provided.

Abstract: Wearable apparatus and method of using same for tracking a labeled probe in a subject are disclosed.

Abstract: Provided are compounds of Formula I, or pharmaceutically acceptable salts thereof, and methods for their use as Nrf2 activators and for their production.

Abstract: The present invention pertains to a cell culture medium comprising copper as a media supplement, which was shown to control recombinant protein glycosylation and methods of sing thereof. The present invention further pertains to a method of controlling or manipulating glycosylation of a recombinant protein of interest in a large scale cell culture.

Abstract: Described herein are compounds represented by formula (I?) or a pharmaceutically acceptable salt thereof, pharmaceutical compositions comprising the same and methods of preparing and using the same. The variables Ar, Ra, Rb, m, n, Y1, Y2, R3 and R4 are defined herein.

Abstract: Disclosed is a cytoprotective agent for use with respect to ischemic damage, including as an active ingredient a triprenyl phenol compound represented by the following general formula (I), wherein X is —CHY—C(CH3)2Z, Y and Z are each independently —H or —OH, or jointly form a single bond, and R represents a hydrogen atom or a substituent with a molecular weight of 1000 or less.