‘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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Orphan drug designation granted to ‘Olaparib’ for pancreatic cancer

U.S. FDA granted orphan drug designation to olaparib for the treatment of patients suffering with cancer of the pancreas. Olaparib (Lynparza, AstraZeneca) is a first-in-class poly ADP-ribose polymerase inhibitor that may exploit inefficiencies of DNA damage response pathway to sabotage cancer cells. In the randomized phase 3 POLO trial, olaparib is being assessed among patients with BRCA-mutated metastatic pancreatic cancer who have not undergone progression following first-line platinum-based chemotherapy.

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Top 10 Hospitals and Doctors for Ovarian Cancer

Ovarian cancer is the cancerous growth in the cells of ovary. This cancer is salient in a way that it often remains elusive until it has spread within the pelvis and abdomen. Ovarian cancer is often referred to as the "cancer that whispers" because its symptoms are vague and inconclusive, and a diagnosis is very seldom made until the cancer has reached advanced stage. Ovarian cancer is diagnosed in an estimated 20000 women in the U.S. each year and the 5-year relative survival rate is 92% if the treatment is started before the cancer has spread outside the ovary (stages IA and IB). To add to the woes, only 15% of all ovarian cancers are found at this early stage. Although it is a highly fatal disease, there is considerable variation in survival, and about one-third of patients survive at least 10 years. The first step in treatment of cancer is selection of doctor and hospital. We, at XpertDox, assayed billions of medically relevant and valid data points to give you the best, data-leveraged and scientific suggestion of the most suitable doctors and hospitals for ovarian cancer.

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Adoptive T-cell Therapy Gives Breakthrough Results in Progressive Multifocal Leukoencephalopathy

Adoptive T-cell therapy has given promising results in a Phase II clinical trial for treatment of progressive multifocal leukoencephalopathy (PML), a rare, life-threatening brain infection with a predilection for patients with cancer and other immune deficiency diseases. The study, held at the Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Centre, showed remarkable improvement in three PML patients infused with T-cells directed at the BK virus.

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Cerdelga offers Sustained Clinical Benefits for Gaucher Patients, Study Shows

Long-term treatment with Cerdelga (eliglustat), for Gaucher disease type 1, is found to be consistently safe. Results of an eight years study proved it to be efficient in causing continuous improvement of various blood parameters, decrease volumes of spleen and liver, and reduce disease activity markers. The study was published in the American Journal of Hematology with the title of “Outcomes after 8 Years of Eliglustat Therapy for Gaucher Disease Type 1: Final Results from the Phase 2 Trial.”

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First New Therapy Approved for X-Linked Hypophosphataemia Patients in 30 Years

NHS has paved way for the children and young adults in England and Wales with the rare bone disorder X-linked hypophosphataemia to have access to a new treatment option. This groundbreaking step has been the first significant ray of hope for these patients in the past 30 years. The National Institute for Health and Care Excellence has given a nod to the use of 'Burosumab' for treating X-linked hypophosphataemia (XLH) in children and young people with growing bones. Burosumab is an anti-FGF23 fully human monoclonal antibody, and has the distinction of being the first treatment to target the pathophysiology of X-linked hypophosphataemia.

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