Rare Disease News and Research RSS Feed - Rare Disease News and Research

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]
Study opens new area of discovery for different aspects of cell biology, biomedical research

Study opens new area of discovery for different aspects of cell biology, biomedical research

Every cell in the body uses phosphorylation, the process of adding a chemical tag to control a protein's function and fate, such as when it moves from one part of a cell to another or binds to other proteins. [More]
PNP Therapeutics granted FDA orphan drug designation for Gedeptin

PNP Therapeutics granted FDA orphan drug designation for Gedeptin

PNP Therapeutics Inc. announced today the Food and Drug Administration has granted orphan drug status to Gedeptin, the Company's lead product candidate (adenoviral vector expressing E. coli purine nucleoside phosphorylase gene) for the intratumoral treatment of anatomically accessible oral and pharyngeal cancers, including cancers of the lip, tongue, gum, floor of mouth, salivary gland and other oral cavities. [More]
Dell extends partnership with TGen to globally expand precision medicine trial for pediatric cancer

Dell extends partnership with TGen to globally expand precision medicine trial for pediatric cancer

Dell today announced its extended partnership with the Translational Genomics Research Institute to help clinical researchers and doctors globally expand the reach and impact of the world's first Food and Drug Administration-approved precision medicine trial for pediatric cancer. [More]
Frankfurt researchers decode molecular details that provide better understanding of neurodegenerative diseases

Frankfurt researchers decode molecular details that provide better understanding of neurodegenerative diseases

Quality control is important - this is not only applicable to industrial production but also true for all life processes. However, whereas an enterprise can start a large-scale recall in case of any doubt, defects in the quality control systems of cells are often fatal. [More]
Mast Therapeutics initiates Phase 3 extension study of vepoloxamer in sickle cell disease

Mast Therapeutics initiates Phase 3 extension study of vepoloxamer in sickle cell disease

Mast Therapeutics, Inc., a clinical-stage biopharmaceutical company leveraging its molecular adhesion and sealant technology (MAST) platform to develop novel therapies for sickle cell disease, heart failure, and arterial disease, today reported that it has initiated an open-label, multicenter Phase 3 extension study of vepoloxamer (MST-188) in sickle cell disease, referred to as "EPIC-E." [More]
Alizé Pharma presents clinical results from the AZP-531 program at the ADA 75th Scientific Sessions

Alizé Pharma presents clinical results from the AZP-531 program at the ADA 75th Scientific Sessions

Detailed data will be presented from the two Phase I trials conducted in 76 healthy volunteers and overweight or obese subjects... [More]
CORD calls upon governments, others to join forces to make Canada's Rare Disease Strategy a reality

CORD calls upon governments, others to join forces to make Canada's Rare Disease Strategy a reality

Today, on Parliament Hill, the Canadian Organization for Rare Disorders released Canada's Rare Disease Strategy and called upon the federal, provincial and territorial governments along researchers, healthcare providers, educators, employers, and the patient community to join forces to make the Strategy a reality. [More]
Lafora rare disease could be aggravated by oxidative stress

Lafora rare disease could be aggravated by oxidative stress

Neurodegenerative Lafora disease usually becomes apparent through seizures during adolescence and puberty and occurs as a consequence of defects in glycogen metabolism and in the cellular mechanisms that are responsible for its disposal. Researchers at the University of Valencia have led a study in which they propose that Lafora rare disease could be aggravated by oxidative stress. [More]
Stanford University professor named recipient of 2015 ECOG-ACRIN Young Investigator Award

Stanford University professor named recipient of 2015 ECOG-ACRIN Young Investigator Award

Heather Wakelee, MD, is the recipient of the 2015 ECOG-ACRIN Young Investigator Award, the ECOG-ACRIN Cancer Research Group announced today. Dr. Wakelee is associate professor of medicine in the Division of Oncology at the Stanford University School of Medicine in Stanford, Calif. [More]
Partners congratulate researchers on forming new biotechnology company

Partners congratulate researchers on forming new biotechnology company

The Children's Hospital of Eastern Ontario, The Ottawa Hospital, the University of Ottawa and McMaster University congratulate its researchers on forming Turnstone Biologics Inc., a biotechnology company focused on developing new treatments for cancer that harness the patient's own immune system. [More]
Advertisement
Advertisement