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FDA clears ArmaGen's AGT-182 IND application for treatment of Hunter syndrome

ArmaGen, Inc., a privately held biotechnology company focused on developing novel therapies to treat severe neurological disorders, announced today that the Investigational New Drug (IND) application for the company's lead product candidate, AGT-182 for the treatment of Hunter syndrome, has been accepted by the U.S. Food and Drug Administration and is now active.

11 Dec 2014

UT Southwestern microbiologists identify key gut bacteria that promotes foodborne infections

UT Southwestern Medical Center microbiologists have identified key bacteria in the gut whose resources are hijacked to spread harmful foodborne E. coli infections and other intestinal illnesses.

11 Dec 2014

Enzymatic activity essential for vision may provide target for drug transport

Researchers at Case Western Reserve University School of Medicine have uncovered the mechanism that enables the enzyme Lecithin: retinol acyltransferase (LRAT) to store vitamin A--a process that is indispensable for vision.

10 Dec 2014

Alizé Pharma completes two Phase I clinical trials of AZP-531 in healthy volunteers, obese subjects

Alizé Pharma SAS, an Alizé Pharma group company specialized in the development of biopharmaceuticals to treat metabolic disorders and rare diseases, announces today the completion of two Phase I clinical trials with its unacylated ghrelin analog AZP-531 in 76 healthy volunteers and overweight or obese subjects.

10 Dec 2014

Simple tips to keep families safe from flu

The flu, or seasonal influenza virus, is extremely unpredictable. Its severity can vary widely from one season to the next depending on many things, including the strains of flu spreading, availability of vaccines, how many people get vaccinated and how well the flu vaccine is matched to the flu viruses circulating each season.

10 Dec 2014

Learning technique may help overcome cognitive issues in fragile X syndrome

A learning technique that maximizes the brain's ability to make and store memories may help overcome cognitive issues seen in fragile X syndrome, a leading form of intellectual disability, according to UC Irvine neurobiologists.

10 Dec 2014

At-home test sniffs out prodromal Parkinson’s disease

An at-home olfactory test helps to identify people with an increased risk of having dopamine transporter imaging indicative of early Parkinson’s disease, show data from the Parkinson Associated Risk Syndrome study.

9 Dec 2014

Scientists receive NIH grants to explore sudden unexpected death in epilepsy

Nine groups of scientists will receive funding totaling $5.9 million in 2014 to work together on increasing the understanding of sudden unexpected death in epilepsy (SUDEP), the leading cause of death from epilepsy. The consortium becomes the second Center Without Walls, an initiative to speed the pace of research on difficult problems in epilepsy by promoting collaborative research.

9 Dec 2014

Kiadis Pharma reports positive interim data from ATIR Phase II clinical study

Kiadis Pharma B.V., a clinical stage biopharmaceutical company developing T-cell immunotherapy treatments for blood cancers, today announces positive interim data from the ongoing Phase II clinical study with its lead product ATIR.

9 Dec 2014

Case Western Reserve selected to lead $27.3 million international effort to identify causes of SUDEP

Case Western Reserve is one of two universities in the country selected to lead a $27.3 million international effort to identify the causes of a mysterious and deadly phenomenon that strikes people with epilepsy without warning.

9 Dec 2014

Amgen announces new data from BLINCYTO Phase 2 study for treatment of patients with ALL

Amgen today announced that new data from a pivotal Phase 2 study evaluating BLINCYTO (blinatumomab) for the treatment of adult patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (ALL) was presented at the 56th American Society of Hematology Annual Meeting and Exposition.

9 Dec 2014

Low doses of fluoxetine could hold key to preventing PMS symptoms

Low doses of fluoxetine - better known as the anti-depressant Prozac - could hold the key to preventing PMS symptoms, an international team of researchers has found.

8 Dec 2014

Researchers report promising outcomes from clinical trial in patients with dyskeratosis congenita

Researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center report promising outcomes from a clinical trial with patients with a rare form of bone marrow failure who received a hematopoietic stem cell transplant (HSCT) after pre-treatment with immunosuppressive drugs only. This is the first trial reporting successful transplant in dyskeratosis congenita (DC) patients without the use of any radiation or conventional cytotoxic chemotherapy beforehand.

8 Dec 2014

Researchers discuss effects of mother-infant bedsharing

Recommendations by physician groups to avoid bedsharing among mothers and their babies are intended to reduce sleep-related infant deaths. But evidence suggests that the risks of bedsharing have been over-emphasized, advice never to bedshare is unrealistic, and avoiding bedsharing may interfere with breastfeeding, according to an article in Breastfeeding Medicine, the official journal of the Academy of Breastfeeding Medicine published by Mary Ann Liebert, Inc., publishers.

8 Dec 2014

Weight-loss surgery may reduce lower urinary tract symptoms

Metabolic syndrome is linked with an increased frequency and severity of lower urinary tract symptoms, but weight loss surgery may lessen these symptoms. The findings, which come from two studies published in BJU International, indicate that urinary problems may be added to the list of issues that can improve with efforts that address altered metabolism.

8 Dec 2014

Two-drug combination may be effective for patients with higher-risk forms of MDS and AML

A phase two study that investigated the potential of the drugs azacitidine (AZA) and lenalidomide (LEN), demonstrated that the two therapies in combination may be an effective frontline treatment regimen for patients with higher-risk forms of myelodysplastic syndrome and acute myeloid leukemia.

8 Dec 2014

Emerging strategies to boost healthy RBCs may reduce burden of anemia linked to blood disorders

Emerging treatment approaches may reduce the burden of anemia associated with blood disorders by enhancing production of healthy red blood cells, according to data presented today at the 56th American Society of Hematology Annual Meeting and Exposition.

7 Dec 2014

Study shows link between cardiac abnormalities, sudden unexpected death in epilepsy

Groundbreaking findings describing the link between cardiac abnormalities and sudden unexpected death in epilepsy (SUDEP) will be presented at the American Epilepsy Society's Annual Meeting in December.

7 Dec 2014

Study provides insights into genetic underpinnings of childhood epilepsies

Technological advances in genetic analysis have uncovered changes in single genes that account for a surprising number of infantile and early-childhood epilepsies. Though some of the affected genes have been identified, the physical manifestations of these alterations remain largely uncharacterized.

7 Dec 2014

Innovative cell therapy shows highly promising results in pediatric patients with ALL

An innovative cell therapy against a highly aggressive form of acute lymphoblastic leukemia (ALL) continues to show highly promising results in children treated in a pilot study. Ninety-two percent of the 39 children receiving bioengineered T cells had no evidence of cancer at one month after treatment, with this complete response persisting in some cases for more than two years.

7 Dec 2014

Syndrome News and Research

FDA clears ArmaGen's AGT-182 IND application for treatment of Hunter syndrome

UT Southwestern microbiologists identify key gut bacteria that promotes foodborne infections

Enzymatic activity essential for vision may provide target for drug transport

Alizé Pharma completes two Phase I clinical trials of AZP-531 in healthy volunteers, obese subjects

Simple tips to keep families safe from flu

Learning technique may help overcome cognitive issues in fragile X syndrome

At-home test sniffs out prodromal Parkinson’s disease

Scientists receive NIH grants to explore sudden unexpected death in epilepsy

Kiadis Pharma reports positive interim data from ATIR Phase II clinical study

Case Western Reserve selected to lead $27.3 million international effort to identify causes of SUDEP

Amgen announces new data from BLINCYTO Phase 2 study for treatment of patients with ALL

Low doses of fluoxetine could hold key to preventing PMS symptoms

Researchers report promising outcomes from clinical trial in patients with dyskeratosis congenita

Researchers discuss effects of mother-infant bedsharing

Weight-loss surgery may reduce lower urinary tract symptoms

Two-drug combination may be effective for patients with higher-risk forms of MDS and AML

Emerging strategies to boost healthy RBCs may reduce burden of anemia linked to blood disorders

Study shows link between cardiac abnormalities, sudden unexpected death in epilepsy

Study provides insights into genetic underpinnings of childhood epilepsies

Innovative cell therapy shows highly promising results in pediatric patients with ALL

Bringing real-time analytics to downstream bioprocess monitoring

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

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