The Current Challenge of Immune Globulin Access
Understanding the factors contributing to the current shortage of immune globulin could help to address a crisis that threatens dire consequences for patients.
Importing Drugs: Is It Safe and Responsible?
As Americans continue to grapple with the skyrocketing costs of prescription drugs, many are turning to other countries to access medications they need at prices they can afford.
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.
Breakthroughs in Stem Cell Research
Optimism continues to grow about the use of stem cells in regenerative medicine highlighted by hundreds of successful clinical trials.
FDA Approves Uplizna to Treat Rare Autoimmune Disease of the CNS
The U.S. Food and Drug Administration (FDA) has approved inebilizumab-cdon (Uplizna) to treat neuromyelitis optica spectrum disorder (NMOSD) in adult patients with the anti-AQP4 antibody.
Rituxan Superior to Mycophenolate Mofetil in Sustaining Complete Remission in Pemphigus Vulgaris Patients
Results from Genentech’s Phase III PEMPHIX study evaluating the efficacy and safety of Rituxan (rituximab) compared to mycophenolate mofetil (MMF) in adults with moderate to severe pemphigus vulgaris (PV) showed the study met the primary endpoint at week 52 and demonstrated Rituxan is superior to MMF.
Large-Dose Albumin Supplementation Does Not Reduce Mortality or Need for Critical Care Interventions: Retrospective Review
Investigators conducted a retrospective review of 38 cases treated at Taipei Veterans General Hospital between January 2007 and December 2018 to determine whether more aggressive albumin supplementation can benefit major burn patients with persistent hypoalbuminemia.
Bleed Protection with up to 14-Day Dosing of Long-Acting Recombinant Factor IX Product in Selected Children with Hemophilia B
In pharmacokinetic studies, a recombinant fusion protein genetically linking human coagulation factor IX with human albumin (rIX-FP) (IDELVION, CSL Behring) has been shown to have an approximately five-fold longer half-life compared with standard recombinant factor IX products.
Measles Virus Destroys Immune System’s Memory of Past Infections
Two studies were conducted to determine whether measles infection causes long-term damage to immune memory.
Study of Kevzara from Severe to Critical COVID-19 Patients
Following a review by the Independent Data Monitoring Committee (IDMC) of preliminary results from the Phase II portion of an ongoing Phase II/III trial evaluating Kevzara (sarilumab), the trial was amended so only critical patients continue to be enrolled to receive Kevzara 400 mg or placebo.
