New Haven, CT – Gaucher disease treatment has come a long way in the past three decades. The United States Food and Drug Administration (FDA) approved the first enzyme replacement therapy (ERT) in 1991. That first approval opened the door for other ERT treatments and paved the road for substrate...
research News
YOGIN, South Korea — GC Biopharma Corp today announces that the US Food and Drug Administration (FDA) has approved ALYGLO (immune globulin intravenous, human-stwk) 10% Liquid, formerly referred to as “GC5107,” for the treatment of adult patients aged 17 years and older with primary humoral immunodeficiency (PI). The ALYGLO pivotal...
Uppsala, Sweden – Women with a type of ovarian cancer known as germ cell tumors have a worse prognosis than men with similar tumours, i.e. testicular cancer. After five years with the disease, 98 percent of men were alive while the survival rate for women was only 85 percent. This...
HOUSTON — As stroma – the supportive framework of the prostate gland – react to prostate cancer, changes in the expression of genes occur that induce the formation of new structures such as blood vessels, nerves and parts of nerves, said researchers at Baylor College of Medicine in a report...
Two new studies from The Children’s Hospital of Philadelphia advance the search for genetic events that result in neuroblastoma, a puzzling, often-deadly type of childhood cancer. Originating in the peripheral nervous system, neuroblastoma is the most common solid cancer of early childhood and causes 15 percent of all childhood cancer...
PHILADELPHIA — Two new studies from The Children’s Hospital of Philadelphia advance the search for genetic events that result in neuroblastoma, a puzzling, often-deadly type of childhood cancer. Originating in the peripheral nervous system, neuroblastoma is the most common solid cancer of early childhood and causes 15 percent of all...
Philadelphia, PA – Immunotherapies that target the CD20 antigen have revolutionized how patients with a variety of blood cancers and hematologic disorders have been treated. However, many patients develop resistance to these treatments due to a loss of the antigen that’s being targeted. Now, a new study from researchers at...
Hemophilia A is an inherited bleeding disease caused by a lack of the blood clotting protein Factor VIII. It had been hoped that gene therapy would provide a breakthrough in treatment, but the most common gene therapy approach has had little clinical success. However, a team of researchers, at the...
Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age. Twenty-two patients were evaluated (ranging in...
Toronto, Canada – When Michael Pirovolakis received an individualized gene therapy in a single-patient clinical trial at The Hospital for Sick Children (SickKids) in March 2022, the course of his condition was dramatically altered. Michael has spastic paraplegia type 50 (SPG50), an “ultra-rare” progressive neurodegenerative disorder that causes developmental delays,...