Toward the Ultimate Cure: Gene Therapy for Severe Combined Immunodeficiency
Gene therapy is the one remaining therapeutic option for treating severe combined immunodeficiency by correcting it at its most fundamental genetic level.
Stem Cell Therapies: The Era of Regenerative Medicine?
Advancements in stem cell therapy may soon eliminate surgeries and cure disease, but first, the debate between two different schools of thought concerning how best to proceed needs to be resolved.
Recombinant Human Soluble Thrombomodulin May Improve Outcomes in Transplant-Associated Thrombotic Microangiopathy
Intravenous administration of recombinant soluble thrombomodulin (rTM) may improve recovery and survival compared to other treatments in patients with transplant-associated thrombotic microangiopathy (TA-TMA) after hematopoietic stem cell transplantation (HSCT), according to a retrospective analysis of 254 consecutive patients at a single Japanese institution.
Transforming Hemophilia Care: A New Generation of Extended Half-Life Factor Concentrates
New extended half-life products represent a powerful new tool for improving the care and quality of life for persons with hemophilia.
Using Genomic Data to Personalize Cancer Treatment
Researchers are using genomics research to better understand how genetic variation contributes to human health and disease.
The New Therapeutic Renaissance for Patients with Rare Bleeding Disorders
As it has since the first commercially produced factor VIII concentrate transformed the lives of U.S. hemophilia A patients, innovation is still the life-blood of this industry.
BabyBIG: Definitive Early Immunotherapy for Infant Botulism
Explore the history and trace the development of BabyBIG, the life-saving immunotherapy for infant botulism.
Precision Medicine: A Seismic Shift in Treatment Strategy
Still in its infancy, precision medicine holds out hope for moving directly from diagnosis to an effective tailor-fit treatment for each individual patient.
The Future Has Arrived: A Wave of New Products Is Redefining Hemophilia Care
New long-acting therapeutics are changing the way hemophilia is managed, creating more choice and better treatment compliance.
Grifols Invests in Alkahest to Develop Plasma-Based Products
Grifols has made a major equity investment in Alkahest to work together to develop novel plasma-based products for the treatment of cognitive decline in aging and disorders of the central nervous system.