Abstract: The disclosure features methods for treating at least one symptom of neurofibromatosis type 1, treating at least one symptom of dystrophic scoliosis, or inducing bone healing by administering a soluble alkaline phosphatase (sALP) to the patient.

Abstract: Disclosed herein are methods for treating lupus nephritis (LN) and/or immunoglobulin A (IgA) nephropathy in a subject. The methods include administering to the subject a therapeutically effective amount of a small molecule complement factor D inhibitor.

Abstract: This disclosure relates to methods of treating a copper metabolism-associated disease or disorder, such as Wilson disease (WD). This disclosure also relates to methods of sequestering copper in a subject or of mobilizing copper into plasma in a subject.

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 provides methods for the synthesis of complement factor D inhibitors and intermediates thereof.

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 present disclosure provides methods for the synthesis of complement factor D inhibitors and intermediates thereof. The methods involve subjecting an intermediate in the synthesis of a complement factor D inhibitor to a t-butyl ester deprotection reaction using a weak base.

Abstract: The disclosure provides engineered polypeptides that specifically bind to human complement component C5 and/or serum albumin. The disclosure also provides fusion proteins comprising such engineered polypeptides, wherein such fusion proteins may be multivalent and multi-specific fusion proteins. The disclosure further provides nucleic acid molecules that encode such engineered polypeptides or fusion proteins, and methods of making such engineered polypeptides or fusion proteins. The disclosure further provides pharmaceutical compositions that comprise such engineered polypeptides or fusion proteins, and methods of treatment using such engineered polypeptides or fusion proteins.

Abstract: The present disclosure relates to antidotes to anticoagulants targeting factor Xa. The antidotes are factor Xa protein derivatives that bind to the factor Xa inhibitors thereby substantially neutralizing them but do not assemble into the prothrombinase complex. In one embodiment, the derivatives described herein lack or have reduced intrinsic coagulant activity.

Abstract: Compositions and methods for treating multiple myeloma (MM), acute myeloid lymphoma (AML) or a myeloproliferative disease (MPD) in a human patient in need thereof. The methods entail administering to the patient an effective amount of cerdulatinib.

Abstract: The present disclosure provides stable liquid pharmaceutical formulations comprising anti-CD200 antibodies or antigen-binding fragments thereof, and articles of manufacture and kits containing the formulations. Also featured are methods of using the formulations in cancer and autoimmune therapies, and for preventing, delaying or treating cell, tissue, or organ transplant rejection.

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

Abstract: The present disclosure is directed to methods of identifying a test compounds for treating sickle cell disease (SCD), ?-thalassemia (BT), or sickle cell BT. The methods involved contacting a test sample including cells with heme, serum, and a test compound, and measuring a biological phenomena including (1) deposition of a complement factor on the cells in the test sample; or (2) effect(s) of the complement factor deposition of (1) on target effector cells, where an attenuation in the biological phenomena in the test sample compared to the biological phenomena in a reference standard is indicative that the test compound is effective in treating sickle cell disease (SCD), -thalassemia (BT), or sickle cell BT.

Abstract: The present disclosure relates to methods, uses, and compositions for the treatment of Sickle cell disease (SCD), beta thalassemia (BT), or sickle cell BT. More specifically, the disclosure concerns the treatment of patients having SCD. BT, or sickle cell BT using a complement C5 inhibitor, such as an anti-C5 antibody or fragment thereof, a nucleic acid molecule, a peptide, a small molecule, or an aptamer.

Abstract: Disclosed are methods of treating a copper metabolism-associated disease or disorder, such as Wilson disease (WD) in a subject, particularly in a subject who is from about 3 years old to less than about 18 years old. Also disclosed are compositions comprising bis-choline tetrathiomolybdate for use in the treatment of a copper metabolism-associated disease or disorder, such as Wilson disease (WD) in a subject, particularly in a subject who is from about 3 years old to less than about 18 years old. Also disclosed are uses of a composition comprising bis-choline tetrathiomolybdate for the manufacture of a medicament for treating a copper metabolism-associated disease or disorder, such as Wilson disease (WD) in a subject, particularly in a subject who is from about 3 years old to less than about 18 years old.

Abstract: The present invention relates to methods, uses, and compositions for the treatment of Sickle cell disease (SCD), beta thalassemia (BT), sickle cell BT. More specifically, the invention concerns the treatment of patients having SCD, BT, or sickle cell BT using a complement pathway component (e.g., Factor P (properdin)) inhibitor, such as an antibody or fragment thereof, a nucleic acid molecule, a peptide, a small molecule, or an aptamer, among others.

Abstract: Featured are pharmaceutical compositions that include a soluble alkaline phosphatase for treating bone mineralization disorders, such as hypophosphatasia (HPP), and symptoms thereof. The polypeptides include a soluble alkaline phosphatase (sALP) or fragment thereof, which is derived from a naturally occurring alkaline phosphatase (ALP).

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 liver function, increasing LAL tissue concentration, and increasing LAL activity in a human patient suffering from LAL deficiency are also provided.

Abstract: Provided are dosages and methods for clinical treatment of dermatomyositis (DM), particularly severe and/or refractory DM, in human patients using an anti C5 antibody, or antigen binding fragment thereof (e.g., such as ravulizumab (ULTOMIRIS®)).

Abstract: Described herein are fusion proteins which include factor H catalytic domains and may include VHH domains, factor H-related protein 5 domains, or integrin binding domains, and the use of such fusion proteins in methods of treatment of diseases mediated by complement alternative pathway activation or dysregulation, for example, kidney diseases.