Ionis completed a Phase 2 clinical study evaluating cimdelirsen (IONIS-GHR-LRx) as an addon therapy in patients with uncontrolled acromegaly despite stable therapy with long-acting somatostatin receptor ligands (SRL). Based on the results of this Phase 2 study and a preliminary analysis of the ongoing open-label study, proof of mechanism was...
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Compared with chemotherapy conditioning, total body irradiation (TBI) may lead to better outcomes prior to allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with acute lymphoblastic leukemia (ALL), according to research in the Journal of Clinical Oncology. Patients with ALL commonly receive TBI and etoposide before undergoing HSCT; however there...
At night, after Tammy Hruskach tucks her boys in bed, she prays a mother’s prayer for constant protection and guidance — first for Logan, 9; and then for her 7-year-old, Austin. “And God,” she always adds as she brushes the bangs off Austin’s forehead, her hand lingering in one last...
BOSTON, Mass. — Therapeutic cancer vaccines are a form of immunotherapy in the making that could not only destroy cancer cells in patients, but keep a cancer from coming back and spreading. Multiple therapeutic cancer vaccines are being studied in clinical trials, but despite their promise, they are not routinely...
IBEC researchers led by Javier Ramón and Juan M. Fernández develop the first three-dimensional model for myotonic dystrophy, a rare disease that currently has no cure. The model combines patient cells and bioengineering techniques and represents a major advance over the use of animals and cell cultures. This new model...
Over 100,000 people suffer from multiple sclerosis in Germany alone. Despite intensive research, the factors that trigger the disease and influence its progress remain unclear. Scientists from the Max Planck Institute of Neurobiology in Martinsried and an international research team have succeeded in attaining three important new insights into the...
BOSTON — Tract Bio, a biotechnology company discovering and developing novel therapies for cancer and inflammatory disease, today announced the publication of a research article in the American Journal of Respiratory and Critical Care Medicine (AJRCCM) highlighting the stem cell variants driving chronic lung inflammation in modulator-treated cystic fibrosis patients...
COLUMBIA, Missouri – As one of the most insidious diseases in the world, cancer has few treatments that work to eradicate it completely. Now, a new ground-breaking approach pioneered by two researchers working at the University of Missouri’s Roy Blunt NextGen Precision Health building shows promising results in preventing lung...
Bottom Line: The MEK inhibitor trametinib (Mekinist) was an effective treatment for pediatric patients with relapsed or refractory juvenile myelomonocytic leukemia (JMML) enrolled in a phase II clinical trial, with seven of 10 patients alive after a median of two years. Journal in Which the Study was Published:...
SAN DIEGO, Calif. — Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to TPN-101 for progressive supranuclear palsy (PSP). “Fast...