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September tip sheet from Cedars-Sinai Medical Center

September tip sheet from Cedars-Sinai Medical Center

A computer algorithm did a better job than doctors at documenting "red flag" symptoms among patients with gastrointestinal disorders, a Cedars-Sinai study has found, underscoring the potential of the technology to improve patient care. [More]

Practices for expediting corporate market research approval process for faster insights

Delays in the launch and execution of critical market research projects can slow down the decision-making process, resulting in missed opportunities and increased costs. It is, therefore, absolutely critical for biopharmaceutical companies to develop a tactical approach to expedite the market research approval process for quicker and more meaningful insights. [More]
GOSH to improve rare disease diagnosis with adoption of Congenica’s Sapientia technology

GOSH to improve rare disease diagnosis with adoption of Congenica’s Sapientia technology

Ian was ‘a clumsy child’. He kept bumping into objects and couldn’t see well in the dark. He endured 15 years of tests and misdiagnoses during a lengthy, and sometimes traumatic, diagnostic odyssey. [More]
FDA, EMA accept filing applications for Boehringer Ingelheim's afatinib to treat patients with advanced SCC of the lung

FDA, EMA accept filing applications for Boehringer Ingelheim's afatinib to treat patients with advanced SCC of the lung

Boehringer Ingelheim today announced that both the U.S. Food and Drug Administration and the European Medicines Agency have accepted filing applications for afatinib for the treatment of patients with advanced squamous cell carcinoma (SCC) of the lung progressing after treatment with first-line chemotherapy. [More]
MTG Biotherapeutics' MTG-201 granted FDA Orphan Drug Designation for malignant mesothelioma treatment

MTG Biotherapeutics' MTG-201 granted FDA Orphan Drug Designation for malignant mesothelioma treatment

MTG Biotherapeutics, an immuno-oncology company developing novel therapeutics for the treatment of cancer, today announced that the US Food and Drug Administration has granted Orphan Drug Designation for MTG-201, an advanced biologic therapy targeting Dickkopf-3 gene defects in various cancers, for the treatment of malignant mesothelioma. [More]
Charlotte charity Taylor's Tale inspires new law to create treatments for rare diseases, spur growth in NC

Charlotte charity Taylor's Tale inspires new law to create treatments for rare diseases, spur growth in NC

Charlotte charity Taylor's Tale helped inspire a law designed to stimulate the creation of new treatments for rare diseases and spur economic development in North Carolina. [More]
Inhibikase Therapeutics launches observational clinical study in patients who survived PML

Inhibikase Therapeutics launches observational clinical study in patients who survived PML

Inhibikase Therapeutics, Inc. announces the launch of an observational clinical study in patients who contracted Progressive Multifocal Leukoencephalopathy (PML), the rare and often fatal brain infection that arises as an unintended side effect of some medications used for treatment of Multiple Sclerosis, Crohn's disease, Ulcerative Colitis and certain cancers of the blood and lymph. [More]
Mallinckrodt subsidiary to acquire Therakos for $1.325 billion

Mallinckrodt subsidiary to acquire Therakos for $1.325 billion

Mallinckrodt plc, a leading specialty biopharmaceutical company, and The Gores Group, a global investment firm, announced today that they have entered into a definitive agreement under which a subsidiary of Mallinckrodt will acquire Therakos, Inc. a leading immunotherapy company in a transaction valued at approximately $1.325 billion. [More]
Vtesse reports preliminary results from VTS-270 Phase 1 trial for treatment of Niemann-Pick Disease Type C

Vtesse reports preliminary results from VTS-270 Phase 1 trial for treatment of Niemann-Pick Disease Type C

Vtesse, Inc. announced preliminary results today from an open-label Phase 1 clinical trial with VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) for treatment of Niemann-Pick Disease Type C (NPC) conducted by researchers at the National Institutes of Health Eunice Kennedy Shriver National Institute of Child Health and Human Development. [More]
European Commission grants orphan drug designation to SGX301 for treatment of CTCL

European Commission grants orphan drug designation to SGX301 for treatment of CTCL

Soligenix, Inc., a late-stage biopharmaceutical company developing products that address unmet medical needs in the areas of inflammation, oncology and biodefense, announced today that the European Commission, acting on the positive recommendation from the European Medicines Agency Committee for Orphan Medicinal Products (COMP), has granted orphan drug designation to synthetic hypericin (the active pharmaceutical ingredient in SGX301) for the treatment of cutaneous T-cell lymphoma (CTCL), a rare disease and a class of non-Hodgkin's lymphoma, a type of cancer of the white blood cells that are an integral part of the immune system. [More]
BGI to hold 10th International Conference on Genomics in October

BGI to hold 10th International Conference on Genomics in October

The 10th International Conference on Genomics (ICG-10) will be held by BGI from October 22-25, 2015, in Shenzhen, China. [More]
Lupin signs strategic asset purchase agreement with Temmler

Lupin signs strategic asset purchase agreement with Temmler

Pharma Major Lupin Limited today announced that it has entered into a strategic asset purchase agreement with TEMMLER PHARMA GMBH & CO. KG, a part of the Aenova Group, one of the world's largest pharmaceutical contract manufacturers, to acquire Temmler's specialty product portfolio subject to certain closing conditions. [More]
Novogen's Anisina granted FDA Orphan Drug Designation for neuroblastoma

Novogen's Anisina granted FDA Orphan Drug Designation for neuroblastoma

US-Australian drug discovery company, Novogen Limited, today announced that it has received notification from the U.S. Food and Drug Administration that its chemotherapy candidate drug, Anisina, has been granted Orphan Drug Designation for neuroblastoma. [More]
Gene therapy used for sight restoration also strengthens visual pathways in the brain

Gene therapy used for sight restoration also strengthens visual pathways in the brain

Since 2007, clinical trials using gene therapy have resulted in often-dramatic sight restoration for dozens of children and adults who were otherwise doomed to blindness. Now, researchers from the Perelman School of Medicine at the University of Pennsylvania and The Children's Hospital of Philadelphia, have found evidence that this sight restoration leads to strengthening of visual pathways in the brain, published this week in Science Translational Medicine. [More]
Canadian researchers launch world's first viral therapy clinical trial to attack and kill cancer cells

Canadian researchers launch world's first viral therapy clinical trial to attack and kill cancer cells

Canadian researchers have launched the world's first clinical trial of a novel investigational therapy that uses a combination of two viruses to attack and kill cancer cells, and stimulate an anti-cancer immune response. Previous research by this team and others worldwide suggests that this approach could be very powerful, and could have fewer side effects than conventional chemotherapy and radiation, although it will take years to rigorously test through this trial and others. [More]
New research identifies potential treatment for primary effusion lymphoma

New research identifies potential treatment for primary effusion lymphoma

New research from the University of Southern California Norris Comprehensive Cancer Center has identified a potential treatment for a rare but previously incurable form of lymphoma that is observed primarily in patients with HIV/AIDS infection. [More]
IRB Barcelona scientists provide molecular details about Seckel Syndrome

IRB Barcelona scientists provide molecular details about Seckel Syndrome

Today in Nature Communications, scientists at the Institute for Research in Biomedicine (IRB Barcelona) provide molecular details about Seckel Syndrome, a rare disease that causes microcephaly, or small brain, and growth delays. [More]
R. Rodney Howell receives ASHG’s annual Advocacy Award

R. Rodney Howell receives ASHG’s annual Advocacy Award

The American Society of Human Genetics (ASHG) has named R. Rodney Howell, MD, Professor in the Department of Pediatrics, Chairman Emeritus of Pediatrics, and Member of the Hussman Institute for Human Genomics, at the University of Miami Leonard M. Miller School of Medicine, as the first recipient of its new, annual Advocacy Award. [More]
Genetic Alliance, Claritas Genomics team up to help patients and families participate in rare disease research

Genetic Alliance, Claritas Genomics team up to help patients and families participate in rare disease research

Today Claritas Genomics and Genetic Alliance announced a partnership to help patients and families find and participate in relevant research opportunities that will advance understanding of individual genetic disorders and development of new therapies. [More]
Pediatric study looks at evidence-based predictors of biphasic allergic reactions

Pediatric study looks at evidence-based predictors of biphasic allergic reactions

Children are more likely to have a repeat, delayed anaphylactic reaction from the same allergic cause, depending on the severity of the initial reaction. The first pediatric study to look at the predictors for this phenomenon was published today in Annals of Allergy, Asthma & Immunology. [More]
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