Treatment with crizanlizumab reduces pain in sickle cell disease patients

Crizanlizumab has been found to reduce pain in patients with sickle cell disease who were facing the brunt of vaso-occlusive crisis (VOC). The vaso-occlusive crisis, or sickle cell crisis, is believed to be ischemic tissue injury caused due to obstruction of blood flow by sickled erythrocytes.

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Empliciti (elotuzumab) granted regulatory approval for Multiple Myeloma

Empliciti (elotuzumab) was granted regulatory approval from the U.S. Food and Drug Administration (FDA) for adults suffering with multiple myeloma who have received at least two therapies in the past. Empliciti, an intravenous treatment, in combination with dexamethasone and pomalidomid as the third-line medication, is a product of Bristol-Myers Squibb.

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EMAPALUMAB-LZSG gets approval by U.S. FDA for Rare Primary Hemophagocytic Lymphohistiocytosis

Emapalumab-lzsg (Gamifant) got approval by the U.S. FDA for the treatment of refractory, recurrent, or progressive form of primary hemophagocytic lymphohistiocytosis in pediatric and adult patients. It also got approval for the cases that experience intolerance to conventional HLH therapy. The interferon gamma blocking antibody is the first approved treatment for this exceedingly rare medical condition that leads to excessive inflammation. Emapalumab grabs the coveted feat of being the first agent targeted to neutralize interferon gamma.

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Low-dose Use of Chemotherapy, Lysodren, May Treat Children with Pituitary Adenoma Who Are Ineligible for Surgery

A study has reported that low-dose Lysodren (mitotane) bears the potential to help restore growth rates and pubertal development, and decrease body mass index (BMI) in children suffering with Cushing’s disease who are not eligible for pituitary surgery. This drug is associated with side effects that need close monitoring due to their toxic potential, the study mentioned. The study was titled as 'Mitotane restores growth and puberty in nine children with Cushing’s disease,' and was published in the journal Endocrine Connections. Lysodren exerts a direct toxic effect on the zona reticularis, which is the innermost layer of the adrenal cortex, responsible for producing cortisol.

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Ribociclib Could be Used for Treatment of Glioblastoma, Study Suggests

A scientific, experimental study has found that the drug ribociclib, recently approved for breast cancer, could be used for the treatment for glioblastoma, one of the most lethal types of cancers in the world today. Glioblastomas are notorious for remaining incurable due to the inherent intricate nature of these tumors, the inability of drugs to penetrate the deep parts of brain tissue, and the lack of synchronized and unbiased correlation between animal models and the human condition. In an innovative phase 0/2 study led by Dr Nader Sanai, the Director of the Ivy Brain Tumor Center at the Barrow Neurological Institute, the drug ribociclib was shown to penetrate the brain tumor properly, providing first evidence of clinically significant activity of this drug in management of human glioblastoma.

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‘Leukine’ (sargramostim) granted Orphan Drug designation for Pulmonary Alveolar Proteinosis

U.S. Food and Drug Administration (FDA) has granted orphan drug designation to 'Leukine' (sargramostim), a product of Partner Therapeutics, Inc., for the treatment of pulmonary alveolar proteinosis (PAP). It is a yeast-derived recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF) that works by causing stimulation of the leucocyte (WBCs) production and promoting their ability to function properly.

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PV-10 gets Orphan Drug Designation for Neuroblastoma

PV-10 (small molecule oncolytic immunotherapy) has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of neuroblastoma, a non-central nervous system pediatric solid tumor. Intratumoral injection of PV-10 has been demonstrated to cause immunogenic cell death in solid tumor cancers. It is also associated with causing stimulation of tumor-specific reactivity in circulating T cells.

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Top 10 Hospitals and Doctors for Leukemia

In plain and simple words, many times, the diagnosis of cancer does more damage than the disease itself. Thankfully, cancer research has made some substantial strokes lately and it is no more a scary ticking bomb. Timely diagnosis and right treatment decisions can rewrite a person’s health destiny. You can outgrow cancer!

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OMS721 granted ‘Orphan Drug’ designation for hematopoietic stem cell transplant-associated thrombotic microangiopathy

Orphan drug designation has been granted to OMS721, a product of Omeros Corporation, from the U.S. Food and Drug Administration (FDA) for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). OMS721 is human monoclonal antibody that targets the enzyme - mannan-binding lectin-associated serine protease-2 (MASP-2). This enzyme has significant governing role in the working of the lectin pathway of the complement system. Phase 3 clinical programs are in progress for OMS721 in atypical hemolytic uremic syndrome (aHUS), in hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) and in immunoglobulin A nephropathy.

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Rare Pediatric Disease designation granted to ‘lonafarnib’ for Hutchinson-Gilford Progeria Syndrome

The drug 'lonafarnib' has been granted Rare Pediatric Disease (RPD) designation by The U.S. Food and Drug Administration (USFDA) for the treatment of both Hutchinson-Gilford Progeria Syndrome (HGPS) and progeroid laminopathies.

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