Genetics & Rare Disease drugs

18 results

  • aldurazyme

    (laronidase)
    Genzyme Corporation
    ALDURAZYME is indicated for treating adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I), and those with moderate to severe symptoms of the Scheie form. Its efficacy in mildly affected patients and on central nervous system manifestations is not established.

  • arcalyst

    (rilonacept)
    Kiniksa Pharmaceuticals (UK), Ltd.
    ARCALYST (rilonacept) is indicated for treating Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome and Muckle-Wells Syndrome, as well as for maintaining remission of Deficiency of Interleukin-1 Receptor Antagonist (DIRA) and treating recurrent pericarditis in patients aged 12 and older.

  • bivigam

    (Immune Globulin Intravenous (Human) 10%)
    ADMA Biologics, Inc.
    BIVIGAM is indicated for treating primary humoral immunodeficiency (PI) in adults and children aged 2 years and older. It addresses conditions such as common variable immunodeficiency, X-linked agammaglobulinemia, congenital agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies.

  • carbaglu

    (Carglumic Acid)
    Recordati Rare Diseases
    CARBAGLU is indicated for adult and pediatric patients as adjunctive therapy and maintenance treatment for acute and chronic hyperammonemia due to N-acetylglutamate Synthase (NAGS) deficiency, as well as adjunctive therapy for acute hyperammonemia caused by Propionic Acidemia or Methylmalonic Acidemia.

  • cholbam

    (Cholic Acid)
    Manchester Pharmaceuticals, LLC
    CHOLBAM is indicated for treating bile acid synthesis disorders due to single enzyme defects and as adjunctive therapy for peroxisomal disorders, including Zellweger spectrum disorders, in patients with liver disease, steatorrhea, or complications from fat-soluble vitamin absorption issues.

  • crestor

    (Rosuvastatin calcium)
    AstraZeneca Pharmaceuticals LP
    CRESTOR is indicated to reduce cardiovascular risk in adults without established coronary heart disease and to lower LDL-C levels in adults and pediatric patients with hyperlipidemia, including those with familial hypercholesterolemia. It is also used for treating primary dysbetalipoproteinemia and hypertriglyceridemia.

  • cutaquig

    (immunoglobulin G)
    Pfizer Laboratories Div Pfizer Inc
    CUTAQUIG is indicated for replacement therapy in primary humoral immunodeficiency in adults and children aged 2 and older. Conditions include common variable immunodeficiency, X-linked agammaglobulinemia, congenital agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies.

  • cuvitru

    (Immune Globulin Subcutaneous (Human))
    Takeda Pharmaceuticals America, Inc.
    CUVITRU is indicated for replacement therapy in primary humoral immunodeficiency (PI) in patients aged two years and older. Conditions treated include common variable immunodeficiency (CVID), X-linked agammaglobulinemia, congenital agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies.

  • cystadane

    (betaine)
    Rare Disease Therapeutics, Inc.
    Cystadane® (betaine anhydrous) is indicated for treating homocystinuria by lowering elevated homocysteine levels. This includes conditions such as cystathionine beta-synthase deficiency, 5,10-methylenetetrahydrofolate reductase deficiency, and cobalamin cofactor metabolism defects.

  • daybue

    (trofinetide)
    Acadia Pharmaceuticals Inc.
    DAYBUE is indicated for the treatment of Rett syndrome in adults and pediatric patients aged 2 years and older.