Abstract: Disclosed herein are methods and compositions for reducing or eliminating a complement-mediated response in a patient receiving treatment for a disease or disorder wherein one or more therapeutic agents is administered to the patient along with one or more complement inhibitors. Administration of the complement inhibitor along with the therapeutic agent results in a reduced or eliminated complement-mediated response, such as a reduction or elimination of symptoms associated with Complement Activation-Related Pseudoallergy (CARPA) or Cytokine Release Syndrome (CRS).

Abstract: This disclosure relates to methods of measuring copper concentrations in biological samples. More particularly, this disclosure relates to methods of measuring non-ceruloplasmin-bound copper concentrations and/or labile-bound copper concentrations in biological samples. Such methods are particularly useful in management and treatment of metabolism-associated diseases or disorders.

Abstract: Provided are methods for clinical treatment of a protein-losing enteropathy, such as lymphangiectasia, using an anti-C5 antibody, or antigen binding fragment thereof, in patients (e.g., pediatric patients).

Abstract: The disclosure relates to methods for treating pemphigus and/or a pemphigoid disease in a subject in need thereof, wherein the methods include administering to a subject in need thereof a therapeutically effective amount of an FcRn inhibitor. In certain embodiments, the FcRn inhibitor is an anti-FcRn antibody or antigen-binding fragment thereof.

Abstract: Disclosed herein are methods and uses of cerdulatinib for treating diseases or conditions, including hematological cancers, and combinations of cerdulatinib for treating such diseases or conditions.

Abstract: The disclosure provides antibodies that are useful for, among other things, inhibiting terminal complement (e.g., the assembly and/or activity of the C5b-9 TCC) and C5a anaphylatoxin-mediated inflammation and, thus, treating complement-associated disorders. The antibodies have a number of improved properties relative to eculizumab, including, e.g., increased serum half-life in a human.

Abstract: Methods for treating Wilson Disease with bis-choline tetrathiomolybdate therapy are provided. The methods may include administering 15 mg or between 30 and 90 mg of bis-choline tetrathiomolybdate once daily to a patient exhibiting NCCcorrected, alanine aminotransferase (ALT), hemoglobin, platelets, or neutrophils levels meeting specified criteria. The methods may include modifying treatment by decreasing or increasing the daily dose of bis-choline tetrathiomolybdate or discontinuing treatment for a period of time.

Abstract: The disclosure provides methods of treating refractory myasthenia gravis (MG) in a subject in need thereof by administering to the subject a substance that specifically binds complement component 5 (C5). In certain embodiments, the substance that specifically binds C5 is a binding protein, such as an anti-C5 antibody. In certain embodiments, the patient achieves and maintains the status of improved or MM according to the MGFA Post-Intervention Status.

Abstract: The present invention is directed to methods of treating multiple myeloma by administering a compound of Formula (I) or pharmaceutically acceptable salt thereof.

Abstract: The present disclosure relates to protein sequences which can be used to generate factor Xa proteins and derivatives thereof. The protein sequences include a factor Xa light chain portion, a heavy chain catalytic domain portion, and an activation peptide C-terminal to the heavy chain catalytic domain portion. It is discovered that when an activation peptide (AP) is fused to the C-terminal end of the heavy chain of the factor Xa protein or derivative, the resulting protein can be more efficiently expressed, and the attachment of the activation peptide (AP) to the heavy chain does not affect the activity of the protein.

Abstract: The present disclosure relates to, inter alia, antibodies, or antigen-binding fragments thereof, that bind to C5a and to use of the antibodies in methods for treating or preventing complement-associated disorders such as, but not limited to, atypical hemolytic uremic syndrome, age-related macular degeneration, rheumatoid arthritis, sepsis, severe burn, antiphospho lipid syndrome, asthma, lupus nephritis, Goodpasture's syndrome, and chronic obstructive pulmonary disease.

Abstract: The disclosure features methods for treating craniosynostosis in a patient (e.g., a patient having hypophosphatasia (HPP) and exhibiting or likely to have increased intracranial pressure (ICP)) by administering a soluble alkaline phosphatase (sALP) to the patient, e.g., in combination with a cranial surgery, e.g., a cranial vault remodeling procedure.

Abstract: The present invention provides methods of treating LAL deficiency comprising administering to a mammal a therapeutically effective amount of lysosomal acid lipase with an effective dosage frequency. Methods of improving growth and river function, increasing LAL tissue concentration, and increasing LAL activity in a human patient suffering from LAL deficiency are also provided.

Abstract: Provided are methods for clinical treatment of Atypical Hemolytic Uremic Syndrome (aHUS) using an anti-C5 antibody, or antigen binding fragment thereof.

Abstract: Provided are methods for clinical treatment of vitiligo using an anti-CS antibody or antigen binding fragment thereof.

Abstract: The present disclosure relates to stable aqueous solutions comprising a high concentration of an anti-C5 antibody (e.g., ravulizumab) and methods for preparing the solutions. The disclosure also provides methods for treating or preventing complement-associated disorders, such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), using the solutions. Also featured were therapeutic kits containing one or more of the solutions and a means for administering the solutions to a patient in need such a treatment.

Abstract: A method for producing a recombinant polypeptide, comprising: (a) providing a 100 L to 25,000 L fed-batch bioreactor comprising (i) cells capable of expressing the recombinant polypeptide asfotase alfa (SEQ ID NO: 1), and (ii) a culture medium suitable for conducting such expression, the culture medium comprising about 25 ?M to about 300 ?M zinc; (b) culturing the cells under conditions suitable to express the recombinant asfotase alfa wherein the pH of the culture medium is about 6.7 to about 7.1, and wherein zinc is added into said culture medium such that the zinc concentration in the culture medium is maintained at a concentration of about 25 ?M to about 300 ?M of zinc.

Abstract: The present disclosure provides processes for the preparation of cerdulatinib, which is of formula I: or a salt thereof. The disclosure also provides intermediates and processes for the preparation of the intermediates useful in the preparation of cerdulatinib or a salt thereof.

Abstract: The present disclosure relates to compositions and methods useful for assessing the efficacy in a patient having suffered from intracranial hemorrhage while undergoing an anticoagulation treatment with a factor Xa (fXa) inhibitor. The method can include administering to the patient a fXa derivative that has reduced catalytic activity as compared to the wild-type fXa protein, is capable of binding to the factor Xa inhibitor and cannot assemble into a prothrombinase complex; obtaining a blood sample from the patient following the administration; and measuring an anti-fXa activity in the sample, wherein the anti-fXa activity reflects the hemostatic efficacy in the patient. Once the assessment is made, suitable medical interventions can be implemented.

Abstract: Provided herein are antibodies that bind to activin receptor—like kinase 2 (ALK2) and are useful for treating bone disorders, such as those involving reduced bone mineral density and bone mineralization defects, and promoting bone growth. Also provided are polynucleotides encoding the antibodies, vectors comprising the polynucleotides, and cells that produce the antibodies.