Hemophilia Gene Therapy: Cures May Finally Be at Hand
The decades-old dream is to correct the factor deficiency through a single infusion of a hemophilia gene therapy that produces steady high protective levels of FVIII or FIX.
Subcutaneous Immune Globulin May Be a Treatment Option in Stiff Person Syndrome Patients Intolerant to Intravenous Immune Globulin
SCIG may be a reasonable and safe alternative for SPS patients who do not tolerate IVIG, with the caveat that allergic and injection site reactions can be a limiting factor for some patients.
Low Rate of Inhibitor Development in Previously Untreated Hemophilia A Patients Managed with Simoctocog Alfa (Nuwiq)
The investigators concluded PUPs treated with simoctocog alfa had a lower high-titer inhibitor rate than PUPs initially treated with hamster-cell-derived recombinant FVIII products.
Against the Storm: High-Dose IVIG as an Immunoregulatory Treatment Strategy for Severe COVID-19
While there may be no “magic bullet” to treat cytokine storm, one widely used immunomodulatory agent in particular — polyclonal intravenous immune globulin (IVIG) purified from healthy donor plasma — is distinguished by the simple fact that it is anything but a narrowly targeted treatment.
Plasma Exchange May Slow Alzheimer’s Disease Cognitive and Functional Decline: Phase 2b/3 Clinical Study Findings
This trial suggests that plasma exchange with albumin replacement could slow cognitive and functional decline in Alzheimer’s disease, although further studies are warranted.
Lower, More Frequent Dosing of IVIG-Dependent CIDP Patients Does Not Improve Efficacy or Safety in CIDP
The investigators concluded lower, more frequent dosing does not further improve the efficacy of IVIG in stable IVIG-dependent CIDP patients, or result in fewer side effects.
The Promise of COVID-19 Hyperimmune Immune Globulin Therapy
As of this writing, NIAID and industry collaborators are also finalizing the design of a large-scale trial to assess whether COVID-Ig can reduce the rate of hospitalization and other medical encounters in earlier-stage COVID-19 patients exhibiting mild to moderate symptoms.
Infection Rates in PI Patients Decline with Increasing IVIG Dosages and IgG Trough Level
Titrating the IgG trough level up to 960 mg/dL progressively reduces the infection rate in PI patients, but there is less incremental benefit above this trough level.
Plasma Therapies: Effective COVID-19 Treatments?
Studies shed light on whether high-dose intravenous immune globulin, convalescent plasma and hyperimmune globulin will be effective therapeutic options for the novel coronavirus.
Stable and Durable Factor VIII Expression in Subjects Dosed with Investigational Hemophilia A Gene Therapy
More than two years after receiving Spark Therapeutics’ investigational adeno-associated virus (AAV)-mediated gene therapy (SPK-8011), five adult subjects with hemophilia A exhibited stable factor VIII (FVIII) expression, with no evidence of FVIII inhibitors or a cellular immune response.
