Recall the saying “there are a thousand ways to skin a cat”? Researchers are rephrasing it in regard to the mosquito. Fifty years ago, we thought that the war against malaria was pretty much won. We had quinine and chloroquine, which were effective against the parasite that causes the disease....
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ORLANDO — Fox Chase Cancer Center investigators report that a two-drug blockade of mTOR signaling appears safe in metastatic kidney cancer in a phase I trial. Early data suggests that a combination of temsirolimus and bryostatin may be active in patients with rare forms of renal cell cancer, which are...
EL PASO — Two-year-old Emmanuel Gonzalez felt the sun on his face for the first time in weeks Sunday. He has a rare immune system disorder and had to stay in his room at Providence Memorial Hospital while he got well enough to make his first trip down to the...
BIRMINGHAM, Ala. – Researchers have discovered the novel mechanism that underlies a previously reported observation that infection by group A Streptococcus bacteria reduces the risk of later developing Type 1 diabetes. The Journal of Immunology reports that vaccination of neonatal mice with group A Streptococcus promoted a clonal expansion of...
MINNEAPOLIS/ST. PAUL — Focal segmental glomerulosclerosis (FSGS) is a rare kidney disorder that affects children and adults, and can lead to kidney failure. New findings from a team led by the University of Minnesota Medical School show patients with FSGS who were treated with the medication sparsentan experienced improved kidney function—making...
PHILADELPHIA – Amicus Therapeutics (Nasdaq: FOLD) announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat for AT-GAA, the Company’s investigational two-component therapy for the treatment of Pompe disease. Pompe disease...
Tick-borne encephalitis (TBE) is a viral infection, endemic to parts of Europe and Asia If approved, the vaccine may help reduce the risk of TBE for people traveling to endemic areas, potentially including military personnel serving in these locations NEW YORK – Pfizer Inc. (NYSE:PFE) today announced that the U.S....
BURLINGTON, Mass. – PharmaEssentia Corporation (TPEx: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, announced that the U.S. Food and Drug Administration (FDA) has approved BESREMi® (ropeginterferon alfa-2b-njft) for the treatment of adults with polycythemia...
SANTA MONICA, Calif. – Kite, a Gilead Company (Nasdaq: GILD), announced the U.S. Food and Drug Administration (FDA) has granted approval for Tecartus® (brexucabtagene autoleucel) for the treatment of adult patients (18 years and older) with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). Following FDA Breakthrough Therapy Designation...
FOSTER CITY, Calif. – Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leader in rare liver disease, announced that the U.S. Food and Drug Administration (FDA) has approved LIVMARLI™ (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older. LIVMARLI, a...