PLEX is often recommended for moderate to aggressive forms of TM and ON, as is very often the case with MOG antibody disease, if there is not much improvement after being treated with intravenous steroids. PLEX is believed to work in autoimmune CNS diseases through the removal of specific or nonspecific soluble factors likely to mediate, be responsible for, or contribute to inflammatory-mediated organ damage. 2,3 An oral steroid taper may be helpful to prevent steroid-withdrawal relapses. Those with MOG antibody disease seem to respond well to steroids. The decision to offer continued steroids or add a new treatment is often based on the clinical course and MRI appearance at the end of 5 days of steroids. The following are possible treatments in the management of an acute event.Īlthough there are no clinical trials that support a unique approach to treat patients with MOG antibody disease, it is well recognized as a standard of care to give high-dose intravenous methylprednisolone for suspected acute myelitis or optic neuritis, generally for 3 to 5 days, unless there are compelling reasons not to. Treatment guidelines for MOG antibody disease have not been established.
COVID-19 and Rare Neuroimmune Disorders.Vega Therapeutics said VGA039 can be a universal hemostatic therapy to treat various bleeding disorders. By attenuating Protein S cofactor function for tissue factor pathway inhibitor α (TFPIα) and activated protein C (aPC), VGA039 augments and restores thrombin generation during both the initiation and propagation phases of coagulation, addressing fundamental deficiencies of clot formation in VWD. VGA039, is the first purpose-built antibody therapy for VWD with a novel mechanism of action targeting Protein S. Current treatments are limited and include factor replacement therapies that require frequent intravenous (IV) infusions. VWD causes severe bleeding that can damage organs and lead to significant impact on patients’ daily lives.
In VWD, defective or low amounts of von Willebrand factor (VWF) lead to insufficient platelet adhesion and unstable clot formation. Von Willebrand disease (VWD) is a bleeding disorder in which the blood does not clot properly. In preclinical studies, VGA039 demonstrated efficacy in VWD, as well as in numerous congenital bleeding disorders. “VGA039 has the potential to be a new treatment for VWD with a profile to reduce the treatment burden for people living with this disease.”Īn ongoing phase 1 clinical study is designed to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of VGA039 in healthy volunteers and VWD patients across sites in the EU and U.S. “Receiving orphan drug designation status is an important step for VGA039,” said Gary Patou, chief medical officer of Vega Therapeutics.
As a subcutaneously self-administered antibody therapy, it has the potential to transform VWD treatment, the company said. By promoting thrombin generation through targeting Protein S, VGA039 addresses a fundamental mechanism of clot formation in VWD. VGA039 is a first-in-class antibody therapy with a novel mechanism of action that modulates Protein S – a key co-factor involved in thrombin generation during both the initiation and propagation phases of coagulation. Vega Therapeutics is a clinical stage biotechnology company developing novel therapies for rare blood disorders. orphan drug designation for its antibody therapy, VGA039, for the treatment of the rare bleeding disorder, von Willebrand disease (VWD). Food and Drug Administration (FDA) has granted Vega Therapeutics, Inc.
0 kommentar(er)
