Abstract: This disclosure relates to crystalline ammonium tetrathiomolybdate having pharmaceutical grade purity and processes for manufacturing crystalline ammonium tetrathiomolybdate. This disclosure also relates to processes for manufacturing bis-choline tetrathiomolybdate having pharmaceutical grade purity.

Abstract: Compositions and methods for treating lymphoma, in particular, T-cell lymphoma and follicular lymphoma, in a human patient are provided. The methods entail administering to the patient an effective amount of cerdulatinib.

Abstract: A bone delivery conjugate having a structure selected from the group consisting of: A) X-Dn-Y-protein-Z; and B) Z-protein-Y-Dn-X, wherein X is absent or is an amino acid sequence of at least one amino acid; Y is absent or is an amino acid sequence of at least one amino acid; Z is absent or is an amino acid sequence of at least one amino acid; and Dn is a poly aspartate wherein n=10 to 16. Compositions comprising same and methods of use thereof.

Abstract: This invention provides, inter alia, a complement-inhibitor-based treatment plan coupled with a risk evaluation and management strategy (“REMS”) and a safety support program (“SSP”) for reinforcing the REMS. The REMS and SPP are implemented using one or more computer devices with software tools programmed to enforce conditions of the REMS and/or prompt follow-ups by registered nurses enrolled in the SSP. The software tool(s) determines whether a prescriber requesting the complement inhibitor has agreed to abide by the REMS, and can prompt a provider of the complement inhibitor to provide updated educational materials to the prescriber at predetermined times or intervals, to monitor the prescriber for compliance with the REMS, and/or to monitor patients for signs of adverse events. Using exemplary embodiments described herein, a risk of adverse events (especially, but not limited to, meningococcal infections) can be managed and an incidence of the adverse events can be reduced.

Abstract: Compositions and methods relating to potentially pathogenic mutations in the nucleotide sequence of a human LIPA gene. Some LIPA gene products have been discovered to be associated with reduced lysosomal acid lipase (LAL) activity.

Abstract: A method of treating a human having a condition or disease related to a bone defect characterized by at least one of: increased level of an alkaline phosphatase ligand, particularly PPi, PLP, or PEA; and decreased alkaline phosphatase activity, compared to a human without said condition or disease, comprising administering to the human a therapeutically effective amount of a polypeptide comprising the amino acid sequence of SEQ ID NO: 1, wherein the polypeptide is administered through at least one subcutaneous injection to the human in a frequency of fewer than three times each week.

Abstract: The present disclosure provides a method of treating seizure in a subject having aberrant alkaline phosphatase activities, comprising administering a therapeutically effective amount of at least one recombinant alkaline phosphatase to the subject.

Abstract: The disclosure features methods for treating hypophosphatasia (HPP) in a patient (e.g., an adult having HPP, such as an adult having pediatric-onset HPP, or an adolescent having HPP) exhibiting decreased pyrophosphate (PPi) or pyridoxal 5?-phosphate (PLP) concentrations in, e.g., a plasma sample, physical impairments, or decreased walking ability by administering a soluble alkaline phosphatase (sALP) to the patient.

Abstract: Described herein are fusion proteins that include two fragments of factor H, a fragment of factor H and an Fc domain, or a fragment of factor H, a fragment of CR2, and an Fc domain. The use of such proteins in methods of treatment for diseases mediated by alternative complement pathway dysmegulation.

Abstract: Provided are methods for clinical treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) comprising administering to the patient an anti-C5 antibody, or antigen binding fragment thereof, wherein the anti-C5 antibody, or antigen binding fragment thereof, is administered (or is for administration) subcutaneously according to a particular clinical dosage regimen (i.e., at a particular dose amount and according to a specific dosing schedule). In one embodiment, the patient has previously been treated with eculizumab (Soliris®).

Abstract: The present disclosure relates to, inter alia, a method of treating age-related macular degeneration (AMD) in a patient, comprising administering an effective amount of a C5 inhibitor or a C5a inhibitor.

Abstract: Provided herein are compositions and methods for treating a human patient with a complement-associated condition (e.g., PNH or aHUS) by subcutaneously co-administering to the patient a hyaluronidase (e.g., rHuPH20) and an anti-C5 antibody, or antigen binding fragment thereof (e.g., ravulizumab).

Abstract: Described herein are isolated monovalent antibodies or antibody fragments thereof that bind human properdin. Such antibodies are useful in methods of treatment for diseases mediated by alternative complement pathway dysregulation.

Abstract: The disclosure features methods for treating or ameliorating at least one symptom of a subject having or being prone to a muscle weakness disease, comprising administering to said subject a therapeutically effective amount of at least one recombinant polypeptide having alkaline phosphatase activity.

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

Abstract: The present invention is related a method of purifying a heterologous protein from an egg white. In some embodiments, the disclosure is directed to a method of purifying a heterologous protein from an egg white comprising the heterologous protein, the method comprising, (a) adjusting the pH of the egg white to a pH of 5.8 to 6.5 to form a pH-adjusted egg white; (b) filtering the pH-adjusted egg white of (a) and collecting a first filtrate; (c) subjecting the first filtrate of (b) to a hydrophobic interaction chromatography matrix, and collecting a first eluate comprising the heterologous protein; (d) adjusting the pH of the first eluate of (c) to a pH of 5.0 to 5.6 to form a pH-adjusted eluate; (e) filtering the pH-adjusted eluate to obtain a second filtrate; (f) adjusting the pH of the second filtrate to a pH of 3.0 to 4.0 to form a pH-adjusted second filtrate; (g) neutralizing the pH-adjusted second filtrate of (f) to a pH of 5.0 to 8.

Abstract: The disclosure features methods for treating tracheobronchomalacia (TBM) in a patient having hypophosphatasia (HPP), such as an infant, by administering a soluble alkaline phosphatase (sALP) to the patient.

Abstract: Provided herein are methods for simultaneously detecting and quantifying antibodies which bind to the same target and have high sequence identity, such as eculizumab and ALXN1210, present together or alone in a biological sample.

Abstract: The disclosure relates to methods for detecting PNH Type II cell populations in biological samples as well as methods for determining whether a patient is at an increased risk for developing thrombocytopenia or thrombosis based on the percentage of PNH Type II cells in the patient's blood. The disclosure also features reagents and conjugates for use in the methods.

Abstract: Provided herein are methods of replicating a large scale eculizumab production cell culture in a small scale culture.